GEN Roundup: Top Trends in Tissue Engineering – Genetic Engineering & Biotechnology News
By Dr. Matthew Watson
References
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2. B. Zhang et al., Biodegradable scaffold with built-in vasculature for organ-on-a-chip engineering and direct surgical anastomosis, Nat. Materials 15, 669678 (2016), doi:10.1038/nmat4570.
3. S. Shukla et al., Progenitor T-cell differentiation from hematopoietic stem cells using Delta-like-4 and VCAM-1, Nat. Methods 14(5), 531-538 (May 2017),doi: 10.1038/nmeth.4258. Epub Apr 10, 2017.
4. M.M. Pakulska, S. Miersch, and M.S. Shoichet, Designer protein delivery: from natural occurring to engineered affinity controlled release systems, Science 351(6279):aac4750, doi: 10.1126/science.aac4750.
5. M.M. Pakulska, C.H. Tator, and M.S. Shoichet, Local delivery of chondroitinase ABC with or without stromal cell-derived factor 1 promotes functional repair in the injured rat spinal cord, Biomaterials (accepted April 2017).
6. TissueGene, TissueGene to Highlight Invossa, the Worlds First Cell-Mediated Gene Therapy for Degenerative Osteoarthritis, at JP Morgan Healthcare Conference, Press Release,accessed June 12, 2017.
7. O.J.L. Rackham et al., A predictive computational framework for direct reprogramming between human cell types, Nat. Genetics 48, 331335 (2016), doi:10.1038/ng.3487.
8. D.B. Kolesky et al., Three-dimensional bioprinting of thick vascularized tissue, Proc. Natl. Acad. Sci. U.S.A. 113 (12), 31793184, doi: 10.1073/pnas.1521342113.
9. M.M. Laronda et al., A Bioprosthetic Ovary Created Using 3D Printed Microporous Scaffolds Restores Ovarian Function in Sterilized Mice, Nat. Commun. 8, 15261 (May 16, 2017).
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GEN Roundup: Top Trends in Tissue Engineering - Genetic Engineering & Biotechnology News
Wheelchair-bound Langley man raising funds for stem cell therapy in Central America – Surrey Now-Leader
By Sykes24Tracey
Logan Van Dyk hopes stem cell operation in Central America will open doors for him ones that shut abruptly on Aug. 3, 2008.
It took seconds for the now 26-year-old Fort Langley residents life to change.
On that day nearly nine years ago, just after he graduated from R.E. Mountain Secondary, Van Dyk suffered a life-altering spinal cord injury in a mountain biking incident.
I was fooling around on a construction site with some friends and I accidentally fell off a dirt pile on my bike, recalled Van Dyk, who was born and raised in Langley. I fell 25 feet and landed on my face.
Van Dyk suffered a spinal cord injury that left him bound to a wheelchair, as a partial quadriplegic.
I got a C56 spinal cord injury, but its incomplete which means theres always a possibility for recovery, Van Dyk said.
Its that hope that has Van Dyk looking to travel to Panama for therapy.
He started doing some research on stem cells and found an institute in Panama City that offers treatments.
Van Dyk sent in an application and on March 8 received an email saying he will make an excellent candidate for stem cell therapy.
Of course there is no guarantee that I will gain anything back but at this point Im willing to try anything to improve the quality of my life, said Van Dyk, who hopes to get into broadcasting.
He has created a GoFundMe page to raise what he believes is the $45,000 necessary to pay for flights, treatments, accommodations, and a personal nurse in Panama City. Visit http://www.gofundme.com/anw8ce-stem-cell-research.
The therapy itself will cost roughly $37,400 US. As well, there are no nurses at the institute who would assist Van Dyk directly.
I receive care twice a day in order to get in and out of my wheelchair and in and out of bed, Van Dyke explained. So I need to get a bit of extra money so I can hire a nurse to come down with me. Im not sure how much that is going to cost.
A friend who works as a nurse guided Van Dyk towards the possibility of travelling to Panama.
She couldnt believe there was nothing that could be done given the circumstances of my injury, Van Dyke said, because Ive got feeling all the way down to my toes. She looked into it and she found this down in Panama City. We looked at it and found some testimonials from some people who have gone down. They say they have about a 75 per cent success rate.
Van Dyk said he always does his best to remain positive and happy in everything he does.
Even the most basic of things take all my effort to achieve, Van Dyk said on his GoFundMe page. There just had to be something out there that could make things easier.
However, time is running out.
Unfortunately, the cutoff is 10 years, Van Dyk said. They wont do this treatment on anybody whose injury is over 10 years old. Im at my deadline.
Positive attitude
After the accident, Van Dyk was told by doctors that it was very unlikely he would ever walk again and would need to be in a power wheelchair for the rest of his life.
Van Dyk said much to their surprise, he wasnt able to accept that and he worked as hard as possible to overcome the barriers the medical world presented him with, and within a few months started using a manual wheelchair every day.
Mobility however, did not return and life has been a constant struggle ever since.
Keeping Van Dyk moving forward has been a positive attitude, and he quickly adapted to his new life in a wheelchair. It was pretty easy to get over it. I never had trouble finding the positives in life. I kind of adapted. A lot of people would say that I am the most positive person that theyve ever met. I get compliments a lot on how well I actually dealt with the injury. It was difficult but it was easy at the same time.
Van Dyk is now turning to the public to help him regain some freedom.
I miss the active lifestyle I led prior to injury, and am getting really tired of sitting all day long with a limited amount of things I can do to entertain myself, Van Dyk said. Im as independent as a I can be and I still rely on a lot of help. If I can get anything back, even just a bit more upper body Im just looking for anything at this point.
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Wheelchair-bound Langley man raising funds for stem cell therapy in Central America - Surrey Now-Leader
Scientists find mechanism behind precise spinal cord development – Medical Xpress
By raymumme
June 29, 2017 A normal developing spinal cord (left) showing precise patterns of gene activity (red, blue, green demarcating different types of cells). In a spinal cord in which one of the signals is disrupted (right) the accuracy of gene activity has been lost. Credit: Anna Kicheva
Scientists have uncovered how nerve cells in the spinal cord are organised in precise patterns during embryo development - a finding that could give insight into regenerative medicine.
As embryos grow and develop they need the right cell types to end up in the right places inside forming organs. This is particularly important in the spinal cord where different nerve cell types must be accurately positioned so that circuits can assemble properly to control muscle movement. But until now the mechanism underlying nerve cell organisation in the spinal cord has remained poorly understood.
In a study published in Science, researchers at the Francis Crick Institute, the Institute of Science and Technology (Austria) and Ecole Polytechnique Fdrale de Lausanne (Switzerland) report that cells destined to become nerve cells in developing mouse embryos use two different signals spreading from opposite sides of the spinal cord - the back and belly side - to measure their position accurately. Based on this map, they turn into the appropriate nerve cell type. The research was funded by the European Research Council and Wellcome.
The team of biologists, physicists and engineers found that the amounts of the two signals originating from the back and belly sides of the body affect gene activity in developing nerve cells. Based on this gene activity in early development, the cells turn into the appropriate nerve cell type for that position in the spinal cord.
"We've made an important step in understanding how the diverse cell types in the spinal cord of a developing embryo are organised in a precise spatial pattern. The quantitative measurements and new experimental techniques we used, as well as the combined effort of biologists, physicists and engineers were key. This allowed us to gain new insight into the exquisite accuracy of embryonic development and revealed that cells have remarkable ability of to orchestrate precise tissue development," says Anna Kicheva, Group Leader at IST Austria.
"We have shed light on the long-standing question of how developing tissues produce the right cells in the right place in the right numbers," says James Briscoe, Group Leader at the Francis Crick Institute. "It's likely that similar strategies are used in other developing tissues and our findings might be relevant to these cases. In the long run this will help inform the use of stem cells in approaches such as tissue engineering and regenerative medicine. However, there is still much more to learn and we need to continue developing these interdisciplinary collaborations to further our biological understanding."
The paper 'Decoding of position in the developing neural tube from antiparallel morphogen gradients' is published in Science.
Explore further: New study reveals how embryonic cells make spinal cord, muscle and bone
More information: "Decoding of position in the developing neural tube from antiparallel morphogen gradients" Science (2017). science.sciencemag.org/cgi/doi/10.1126/science.aam5887
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Scientists find mechanism behind precise spinal cord development - Medical Xpress
Growth Hormone and Rehabilitation Promoted Distal Innervation in a Child Affected by Caudal Regression Syndrome … – UroToday
By NEVAGiles23
We treated a 9-month old child affected by caudal regression syndrome (CRS) as we treat patients with spinal cord injuries. His spinal cord had been interrupted during fetal development at the L2-L3 level, therefore no innervation (sensitive or motor) existed beyond this level: flaccid paraplegia of inferior limbs and clubfoot, and neurogenic bladder and bowel. Moreover, there was sacral agenesis and right renal agenesis.
Treatment consisted in Growth Hormone (GH) administration plus two daily sessions of specific physiotherapy. GH treatment was given 5 days/week during 3-months followed by 15-days without GH administration; this protocol was repeated during 5 years, being GH doses periodically adjusted to the weight of the patient.
Changes observed in the child were assessed by carrying out the GMFM-88 test and evaluating sensitive and motor ASIA scores. GMFM-88 score at admission was 12.31%, while sensitive ASIA score was 168, and motor ASIA score was 50. Sensitive innervation began to appear quite earlier than motor innervation and reached the maximum ASIA score (224) two years after the treatment commenced. At this time a pelvic floor therapy was added (1 session/week) to rehabilitation. One year later the patient began to walk with crutches; then melatonin was given, at a daily dose of 50 mg (before going to bed) for counteracting the increased production of oxygen free radicals due to the physical effort induced by walking without the support of sacrum and the existence of hip luxation.
Five years after the treatment commenced the GMFM-88 test reached a score of 78.38% (maximum value: 100), while ASIA motor score was 84 (maximum value: 100). Full control of sphincters has been achieved.
To our knowledge this is the first case in which highly significant improvements have been obtained in this syndrome, until now considered to be irreversible. Most likely the early treatment with GH and rehabilitation was the factor responsable for the improvements observed. Since GH has been the only variable we introduced with regard to usual rehabilitation therapies, we think that the hormone induced the proliferation and differentiation of SC ependymal stem cells that led to the formation of a net of new specific nervous connections (perhaps arising from the last existing spinal nerve), although only a tractography may explain what was the origin of the new innervation, since the last MRI study performed when the child was 5-years old did not reveal any change in the vertebral columna and SC with regard to the first MRI study (7-days old). No adverse effects were observed during the treatent with GH and melatonin. Figure 1.3D reconstruction of a CT-SCAN. Age 4-years. It can be seen where the SC interrupted its development (hypoplastic L3), the existence of sacral agenesis, the articulation of iliac bones and the rotation of the left hip. Figure 2. 3-months after the treatment began. Note the abnormal position of the legs and feet. The child only could move his arms and trunk. Figure 3.1 year of treatment. Note the position of his feet. Sensitivity existed but he only could move by crawling with his arms and trunk.
Figure 4.After 2-years of treatment full sensitivity existed. In the image the child is signaling where he had been touched (his eyes were covered with a pillow.
Figure 5.4-years of treatment. The child is able to make plantar flexion (against resistence) and dorsiflexion (not showed) with both feet.
Figure 6.5-years of treatment. The child is able to get up from the floor and keep standing with arms outstretched.
Written By:Jess Devesa, MD, PhD,Scientific Direction, Medical Center Foltra, 15886 Teo, Spain
Read the Abstract
Human head transplant: Chinese researchers claim success with rats – Alphr
By NEVAGiles23
A quick glance at your calendar will reveal that we're now in 2017. 2017, you may recall, is the year when contraversial surgeon Sergio Canavero has promised to perform the world's first human head transplant.
But just how feasible is a human head transplant? Is it the stuff of science fiction, or does it have a basis in current scientific thinking? Read on for everything you need to know about 2017 most alarming scientific development.
The most recent news comes from the journal CNS Neuroscience and Therapeutics, where Xiaping Ren from the Harbin Medical University claims to have successfully repaired severed spinal cords in rats using the same principals that Dr Canavero wants to use on humans before the year is out.
Nine rats were treated with polyethylene glycol (PEG). Eight were still alive a month after the operation, and by day 28 they had regained the ability to walk two were described as being "basically normal".
Canavero told Newsweek that this showed his critics were wrong: "Critics said the transected spinal cord is unrecoverable and thus a human head transplant is impossible...The scans show the reconstructed cord. No pain syndrome emerged over the duration of the study, again rebutting a critics worse than death remark."
Time will tell the team plans to move onto dogs next. It shoud be noted, however, that even if this is right and even if it is applicable to humans, this relates to repairing severed spinal cords not attaching a new head. If the research is accurate, it is indeed a point for Canavero and his proposed methods but we're a long way from it being anything close to the finished article.
A human head transplant is exactly what it sounds like taking one living head and putting it onto a new body.
But actually, thats a little misleading. In real terms, its a body transplant, as the head will be gaining a new body to control. However, as the term whole body transplant is already used to mean transferring the brain between bodies, calling it a head transplant makes it clear that the whole head is to be switched, brain included.
Until recently, a head transplant seemed totally implausible, but the Italian scientist Dr Sergio Canavero believes its possible, and intends to conduct the first surgery in 2017.
Canavero outlines the procedure in detail here, but these are the basics of the process. Remember: dont try this at home, kids.
The donor body and the head to be attached are first cooled down to 12-15C to ensure that the cells last longer than a few minutes without oxygen. The tissue around the neck is then cut, with the major blood vessels linked with tiny tubes. The spinal cord on each party is then severed cleanly with an extremely sharp blade.
"Post coma, Canavero believes the patient would immediately be able to move, feel their face and even speak with the same voice."
At this point, the head is ready to be moved, and the two ends of the spinal cord are fused using a chemical called polyethylene glycol, encouraging the cells to mesh. This chemical has been shown to prompt the growth of spinal cord nerves in animals, although Canavero suggests that introducing stem cells or olfactory ensheathing cells into the spinal cord could also be tried.
After the muscles and blood supply are successfully connected, the patient is kept in a coma for a month to limit movement of the newly fused neck, while electrodes stimulate the spinal cord to strengthen its new connections.
Following the coma, Canavero anticipates that the patient would immediately be able to move, feel their face and even speak with the same voice. He believes physiotherapy would allow the patient to walk within a year.
He explains his suggested methods in the TED talk below.
Sceptical would be a nice way of putting it. Horrified would, in most cases, be more accurate.
Dr Hunt Batjer has attracted headlines for being particularly blunt: I would not wish this on anyone. I would not allow anyone to do it to me as there are a lot of things worse than death.
Dr Jerry Silver witnessed the 1970s monkey head transplant experiment more on which later and describes the procedure as bad science, adding that just to do the experiments is unethical. This is a particular blow to Canavero, as he states that Silvers own work in reconnecting rats spinal cords should give hope to the human head transplant. Silver dismisses this: To sever a head and even contemplate the possibility of gluing axons back properly across the lesion to their neighbours is pure and utter fantasy in my opinion.
Dr Chad Gordon, professor of plastic and reconstructive surgery and neurological surgery at Johns Hopkins University, agrees that Canaveros claims are scientifically implausible. He told BuzzFeed: Theres no way hes going to hook up somebodys brain to someones spinal cord and have them be functional.
On the conservative side, were about 100 years away from being able to figure this out, he continued. If hes saying two, and hes promising a living, breathing, talking, moving human being? Hes lying.
Dr Paul Myers, associate professor of biology at the University of Minnesota at Morris, puts it even more explicitly: This procedure will not work... Try it with monkeys first. But he cant: the result would be, at best, a shambling horror, an animal driven mad with pain and terror, crippled and whimpering, and a poor advertisement for his experiment. And most likely what hed have is a collection of corpses that suffered briefly before expiring.
Others wonder whether Canavero might simply be enjoying the limelight with a PR stunt, including Dr Arthur Caplan, director of ethics at the NYU Langone Medical Centre. Describing the doctor as nuts, he explained to CNN: Their bodies would end up being overwhelmed with different pathways and chemistry than theyre used to, and theyd go crazy.
"We'll probably see a head on a robot before we see it on [another] body," he told Live Science.
Dr John Adler of Stanford University's school of medicine is slightly more optimistic... but not much more. "Conceptually, much of this could work, but the most favourable outcome will be little more than a Christopher Reeve level of function," he told Newsweek.
Canavero is aware of this criticism, claiming that silently hes received a lot of support from the medical community. Of Dr Batjers comments that the surgery would be a fate worse than death, Canavero is scathing. Hes a vascular surgeon. A vascular surgeon of the brain, yes, but he knows nothing, he argued. How can you say such a thing? Its incredible.
"The world is moving, the critics are dwindling. Of course, there will always be critics. Science teaches us that when you propose something groundbreaking, you must be confronted by criticism. If no critics really step forward, you are saying nothing special," he told Medical News Today.
Dr Canavero also believes that the operation could essentially be used to revive the dead, if brains were suitably frozen and stored. In an interview with German magazine Ooom, Canavero said: "We will try to bring the first of the company's patients back to life, not in 100 years. As soon as the first human head transplant has taken place, i.e. no later than 2018, we will be able to attempt to reawaken the first frozen head.We are currently planning the world's first brain transplant, and I consider it realistic that we will be ready in three years at the latest."
No-one has ever attempted a human head transplant before, and attempts on animals have to put it charitably had limited success.
Image: from Motherboard, uploaded under fair use from a 1959 issue of Life
The photo above really does show a dog with two heads and its not a fake. This was the work of Soviet scientist Vladimir Demikhov, and for four days the hybrid of two dogs lived as normally as such a scientific horror could be expected to. Then they died.
Demikhov tried the experiment more than 24 times, but was unable to find a way of avoiding the dogs dying shortly after surgery. Although the results are horrifying to see, Demikhovs research did pave the way for human organ transplants.
"For four days this hybrid of two dogs lived as normally as such a scientific horror could be expected to. Then they died."
But back to the topic of head transplants. The first time a straight swap was successful, was by Dr Robert White, in an experiment on a rhesus monkey in 1970. I feel the need to qualify the word successful with quotation marks, because although the monkey did live, he didnt live very long. Eight days, to be exact, and as the spinal cord wasnt attached to its new body, the monkey was paralysed for its remaining days. However, it could indeed see, hear, smell and taste before the body rejected the foreign head.
According to Canavero in his paper on human head transplants, the monkey lived eight days and was, by all measures, normal, having suffered no complications. However, Dr Jerry Silver who worked in the same lab as Dr White has more haunting memories. He toldCBS: I remember that the head would wake up, the facial expressions looked like terrible pain and confusion and anxiety in the animal. The head will stay alive, but not very long. It was just awful. I dont think it should ever be done again.
More recently, Chinese doctor Xiaoping Ren claims to have conducted head transplants on more than 1,000 mice. The Wall Street Journal reports to have witnessed a mouse with a new head moving, breathing, looking around and drinking. But, crucially, none of these mice have lived longer than a few minutes.
Still, Dr Rens studies continue, and the latest reports are said to be promising, offering a possible answer to the risk of severe blood loss (or brain ischemia) during transplantation. The experimental method that we have described can allow for long-term survival, and thus assessment of transplant rejection and central nervous system recovery, bringing us one step closer to AHBR in man, the researchers wrote.
Ren himself has not ruled out taking part in the first human head transplant operation, according to the Daily Mail. "A human head transplant will be a new frontier in science. Some people say it is the last frontier in medicine. It is a very sensitive and very controversial subject but if we can translate it to clinical practice, we can save a lot of lives," he said.
"Many people say a head transplant is not ethical. But what is the essence of a person? A person is the brain not the body. The body is just an organ," he added.
In January 2016, Canavero told New Scientist that a head transplant had been successfully completed on a monkey in China, although details were sparse. "The monkey fully survived the procedure without any neurological injury of whatever kind," he said, although the article notes that the monkey only kept alive for 20 hours after the surgery for "ethical reasons," limiting its use as a comparison somewhat.
In September 2016, Canavero revealeda further trial of the head transplant on dogs.New Scientisthas seen video footage of a dog appearing to walk three weeks after its spinal cord was severed, with Canavero claiming that the outcome is the result of the same techniques he plans to use on Spiridonov next year.
However, speaking to a number of scientists for their view on the new evidence, New Scientistcould find few sceptics converted. "These papers do not support moving forward in humans," said Jerry Silver a neuroscientist at Cape Western Reserve University in Ohio.
"The dog is a case report, and you cant learn very much from a single animal without controls. They claim they cut the cervical cord 90 per cent but theres no evidence of that in the paper, just some crude pictures," added Silver.
In May 2017, Canavero claimed success with another animal model: rats. Canavero and his team of Chinese surgeons claimed they were able to transplant the head of a donor rat onto the back of a larger one, creating a two-headed animal. The creature's donor head was allegedly able to blink and respond after the operation, although it only lived for 36 hours, which may not inspire confidence even with rodents' reduced lifespans.
You could say so, though Canavero doesn't see it quite like that. In fact, controversially he sees it more as a failure of other types of medicine, telling Medical News Today, "It will be about curing incurable neurological disorders for which other treatments have failed big time, so gene therapy,stem cells- they all just came to nothing. We have failed despite billions of dollars being poured into this sort of research."
"So actually, head transplant or body transplant, whatever your angle is, is actually a failure of medicine. It is not a brilliant success, a brilliant advancement to medical science. When you just haven't tackled biology, you don't know how to treat genes, you don't really understand, and you really need to resort to a body transplant, it means that you've failed. So this must not be construed as a success of medical research," he added.
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Human head transplant: Chinese researchers claim success with rats - Alphr
He broke ground in stem-cell research. Now he’s running for Congress. – Washington Post
By daniellenierenberg
The small pack of scientists running for political office has grown by one.
Stem-cell researcher Hans Keirstead, 50, announced last week that he will try to unseat Californias Rep. Dana Rohrabacher (R). Keirstead, a Democrat with a PhD in neuroscience from the University of British Columbia, was a professor at the University of California at Irvinebefore launching and selling several biotech companies.
Rohrabacher, who represents the 48th District in Southern California, has been in Congress since 1988. Democrats there see 2018 asa vulnerable year for the incumbent. Although Republicans outnumber Democrats in thedistrict, Hillary Clinton swung it in the 2016 election. And Rohrabacher has come under scrutiny for his support of acloser relationship with Russia. In May, the chair of Orange County Democrats toldThe Washington Post that challengers were coming out the woodwork to oppose him. Five candidatesbesides Keirstead have declared they are running for the seat.
Keirstead emerged from academic and entrepreneurial fields. Hepioneered a technique to purify stem cells You cant go putting toenails into the spinal cord, he said and applied this method to spinal-cord injuries and diseases such ascancer and amyotrophic lateral sclerosis, or ALS. In 2014,he sold a stem-cell company in a deal reportedly worth more than $100 million. (He will not fundhis own campaign, he told the Los Angeles Times.) Keirstead has thesupportof314 Action, a nonprofit group that encourages scientists to seek public office.
The Post spoke by phone with the first-time candidate. The following is lightly edited for space and clarity.
TWP: Your opponent, who is a member of the House Science Committee, told Science magazine in 2012 that he loved science. How would you compare your approaches to science?
Keirstead:Im delighted that Dana Rohrabacher loves science. Thats fabulous. But Im also very convinced that he doesnt understand science. Theres a real big difference. If you love science, thats one thing. If you dont understand it, you cant effect change, and you make wrong decisions.
Dana Rohrabacher does not understand global warming. He actually attributed it to the flatulence of dinosaurs, in a serious manner, a while back. [Rohrabacher hassaid this wasa joketo make fun of scientists who study cow methane.]
His inaction and lack of understanding has tremendous detriment on the scientific community. Likewise is the funding to health care and how to fix the health-care system that [former president Barack] Obama put in place. That was not a perfect system by any means; its got problems.But it has also bettered our system. It needs to be worked with in order to further better our system.
TWP: Has your career in stem-cell research influenced your politics?
Keirstead:I was front and center in the national and international debate on stem cells. I was the first scientist in the world to have developed a treatment for spinal-cord injury using stem cells. The dramatic nature of the recovery we saw in rodents, going from paralyzed to walking, drew a great deal of attention and really put me at the center of this issue as it was just coming to light in the public forums.
I did a lot of advising of senators and congressmen all throughout those years and periodically since that time. . . . I was one of the key scientific advisers to Proposition 71 that turned into the $3 billion California Institute of Regenerative Medicine, a not-for-profit that distributes $300 million every year for regenerative medicine in a broad sense.
That was a very good example of how medical breakthroughs and discoveries and advancement are not at odds with economic development. You do not have to cut medical budgets to stimulate the economy. Any scientist and medical doctor will tell you: Give me some time, and I will generate a treatment. And most of the time they are right. What happens with that treatment is small companies are born, people stop dying, quality of life improves.
I see what the governments doing right now as very much opposite that. Frankly, when I look at the deficits of Congress, I see why. When I look at who is in the administration, the types of individuals that we have in Congress, I see very hard-working people doing what they feel is a terrific job. But there is just not the broad and deep field experience in the medical and health-care sectors.
TWP: Was it this perceived deficit that motivated you to run for Congress?
Keirstead:First and foremost, I see it as a continuation of my lifelong pursuits of trying to help people.
I see Congress as a larger stage to effect positive change. If I could have some positive influence in Congress, I could aid [those] that are trying to do good in the world but are having difficulty.
Let me give you an example: Im now expanding into brain cancer. Im running a Phase 2clinical trial with my team.I will not be able to do that if these policy changes of Trumps are instituted and a small company like mine is faced with double user fees. Its not in the budget. I cant ask an investor for another half of a million dollars for an administrative fee.
I see the administration putting insurmountable challenges in front of small businesses. Im about generating treatments to help people, putting medicines in peoples homes. And Im looking to the future and seeing that tap shut off.
Read more:
As scientists erupt in protest, a volcanologist runs for Congress
This group wants to fight anti-science rhetoric by getting scientists to run for office
Tens of thousands marched for science. Now what?
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He broke ground in stem-cell research. Now he's running for Congress. - Washington Post
Spinal cord | Define Spinal cord at Dictionary.com
By LizaAVILA
Contemporary Examples
Infections can strike joints, airways, the lungs, the brain and the tissues lining the spinal cord, or the bloodstream.
Other spinal cord regeneration efforts involve using stem cells to regrow damaged or lost neurons.
As a result, Smith lost his right arm underneath the elbow and parts of his leg, hip and spinal cord.
Generally, a human will only be infected if they eat the nerve tissuebrains or spinal cordof an infected animal.
The bulleta live fire bulletexited through his back but not before severing his spinal cord.
British Dictionary definitions for spinal cord Expand
the thick cord of nerve tissue within the spinal canal, which in man gives rise to 31 pairs of spinal nerves, and together with the brain forms the central nervous system
spinal cord in Medicine Expand
spinal cord n. The thick, whitish cord of nerve tissue that extends from the medulla oblongata down through the spinal column and from which the spinal nerves branch off to various parts of the body. Also called spinal marrow.
spinal cord in Science Expand
The long, cordlike part of the central nervous system that is enclosed within the vertebral column (spine) and descends from the base of the brain, with which it is continuous. The spinal cord branches to form the nerves that convey motor and sensory impulses to and from the tissues of the body.
spinal cord in Culture Expand
The thick column of nerve tissue that extends from the base of the brain about two thirds of the way down the backbone. As part of the central nervous system, the spinal cord carries impulses back and forth between the brain and other parts of the body through a network of nerves that extend out from it like branches.
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Spinal cord | Define Spinal cord at Dictionary.com
Familial ALS Linked to Both Neuron and Astrocyte Pathology – Alzforum
By LizaAVILA
17 Jun 2017
Animal models and engineered cells have contributed their fair share of insight into the pathogenesis of amyotrophic lateral sclerosis (ALS), but researchers do not often have the opportunity to peer into afflicted human neurons. In a study published May 30 in Cell Reports, researchers led by Rickie Patani and Sonia Gandhi at University College London offer a new model for human disease by using motoneurons and astrocytes derived from amyotrophic lateral sclerosis (ALS) patient stemcells.
The researchers generated spinal motoneurons and astrocytes from induced pluripotent stem cells (iPSCs) of ALS patients carrying mutations in the valosin-containing protein (VCP) gene. VCP mutations account for 2 percent of familial ALS cases and have been linked to other disorders as well, including hereditary inclusion body myopathy with Paget disease of bone and frontotemporal dementia (IBMPFD), jointly termed multisystemproteinopathy.
Previously, researchers reported that VCP mutations promoted the formation of cytoplasmic aggregates of TDP-43,one of the hallmarks of motoneuron disease, and that these aggregates were at least partially responsible for the VCP-induced neurodegeneration seen in a fly model (Neumann et al., 2007; Ritson et al., 2010).
Few studies have tested what VCP mutations do to human neurons, however. Using an iPSC-derived neuronal model, Virginia Kimonis at the University of California, Irvine, found that the VCP R155H mutation boosted cells levels of TDP-43 and several proteins involved in protein disposal (Dec et al., 2014).
In search of a more complete view of how VCPs effects unfold over time, first co-authors Claire Hall, Zhi Yao, Minee Choi, and Giulia Tyzach used iPSC-derived populations of spinal cord motoneurons and astrocytes to track several cellular functions. The cells originated from two patients (four clones) with VCP mutations and three healthycontrols.
TDP-43 (red) stays in the nucleus (blue) in control motoneurons (left) but leaks into the cytoplasm in three-day old VCP-mutant motoneurons (right). [Cell Reports, Hall et al.,2017.]
More VCP-mutant motoneurons than controls died; they also made fewer synapses with their neighbors, and had less intense and fewer coordinated bursts of firing than controls. Monitoring the whereabouts of TDP-43, the authors found no difference between mutant and normal cells when the cells were at an early stage of differentiation, as neural precursor cells. But by the third day after becoming motoneuron-like, the mutant cells were leaking TDP-43 from their nuclei into the cytoplasm (see image above). At the same time, they had increased levels of markers of endoplasmic reticulum (ER) stress, became less able than controls to survive an ER stress assay, and began producing reactive oxygen species (ROS) at a higherrate.
By day 17, the mutant motoneurons had sickly ER tubules. Some were swollen, others cozied up to mitochondria, both signs of continued ER stress. The neurons also had low mitochondrial membrane potential and low glutathione levels, indicating mitochondrial dysfunction and oxidative stress,respectively.
Putting these findings together, Patani and Gandhi suggested that cyotosolic TDP-43 generates ER stress, which next triggers increased tethering of the ER to mitochondria. This could depolarize mitochondria, which could impair mitochondrial function and ultimately lead to high levels of free radical production and oxidative stress.The findings support a role for TDP-43 wreaking havoc in the cytoplasm, saidPatani.
The mechanism that links TDP-43 and ER stress remains uncertain, said Kimonis. Patani and Gandhi acknowledge the link is probably complex, noting that, in addition to cyotplasmic TDP-43 aggregates inducing ER stress, ER stress itself seems to drive TDP-43 leakage (Walker et al., 2013).
The researchers then probed how mutant VCP might affect astrocytes. Studies of astrocytes in other ALS models have yielded varying results depending on the mutation. Superoxide dismutase 1 (SOD1) mutations, for example, can turn astrocytes into motoneuron killers (see Oct 2011 news),but it is unclear if they endanger the survival of the astrocytes themselves. Conversely, mutations in TDP-43 seem to sicken astrocytes without making them toxic to neighboring neurons (Serio et al., 2013).
On the left, healthy astrocytes (red) protect VCP-mutant motoneurons (yellow) from death (green). VCP-mutant astrocytes are much less protective (right). [Courtesy of ZhiYao.]
Interestingly, Patani and Gandhi found that VCP mutations had both cell-autonomous and non-cell-autonomous effects. They increased astrocytes risk of death and rendered them less able to support the survival of both control and VCP-mutant motoneurons (see image at right). The authors also found that the cell-autonomous effects on astrocytes differed from those in motoneurons. VCP-mutant astrocytes seemed more resistant when challenged with an ER stressor and had only a transient drop in mitochondrial membrane potential and a transient increase in ROS, with no change in glutathionelevels.
The study is a validation of key prior findings using iPS-derived neurons and glia from patients, which is a nice advance, wrote Paul Taylor at St. Jude Childrens Research Hospital in Memphis, Tennessee. The characterization of the role of glia is also interesting and potentially important.
Looking ahead, Patani and Gandhi want to generate isogenic controls, that is, cells derived from the same patients but with repaired VCP mutations. They also want to create iPSC-derived upper motoneurons, and to better understand muscle pathology, Kimonis hopes to extend her investigation of iPSC-derived muscle cells (Llewellyn et al., 2017).
Other model systems will be of value, too. iPSC-derived models are not equivalent to the decades-old cells inhabiting an adult nervous system. A study of dopaminergic neurons, for example, revealed that gene expression and DNA methylation patterns differed between iPSC-derived neurons and their in vivo counterparts (Roessler et al., 2014).
In the long term, Patani hopes that shedding light on the sequence of events that marks motoneuron degeneration in human cells will enable researchers to more effectively search for ALS therapies.MarinaChicurel
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Familial ALS Linked to Both Neuron and Astrocyte Pathology - Alzforum
Eva Feldman steps down as Taubman Institute director – The Detroit News
By JoanneRUSSELL25
Eva Feldman, M.D., Ph.D.(Photo: Detroit News file photo)
World-renowned researcher Dr. Eva Feldman is stepping down as director of the University of Michigans A. Alfred Taubman Medical Research Institute, an organization that supports research for cures for debilitating and deadly diseases.
Feldman was the founding director of the institute launched by philanthropist and businessman A. Alfred Taubman, who died in 2015 at age 91. Feldman spent the last 10 years at the helm as the institute developed new drugs, surgeries and therapies for diseases such as adult and childhood cancer, diabetes, cardiovascular disease and, Feldmans research interest, amyotrophic lateral sclerosis.
When Alfred and I began the institute 10 years ago together, we had a vision of creating an institute where clinician scientists would take their most novel discoveries from their laboratory and translate that into patient clinical trials, said Feldman, adding that the team of four has grown to over 200. Weve really been able to realize that vision.
Feldman, 65, said Thursday that she raised the institute from its infancy.
Its now time for the next person to take it through its adolescence, because its still growing, she said. So it just seemed the perfect time to pass the baton to someone to have the next 10 best years of their life the way I just had the 10 best years of my life.
The institute will select the next director soon, Feldman said.
A Russell N. DeJong Professor of Neurology, Feldman will continue to run the ALS clinic at UM and her own laboratory, Program for Neurology Research & Discovery. Her team of 30 scientists have spent years working on human clinical trials that may lead to a treatment for ALS, a neurological disease also known as Lou Gehrigs disease. Most ALS patients lose the ability to walk and talk, and death may occur within three to five years of diagnosis.
In 2010, Feldman started the first human clinical trial approved by the U.S. Food and Drug Administration to use stem cells to treat ALS. The FDA recently approved Feldman to move onto the final, nationwide phase of the trial that involves injecting stem cells into the spinal cord of ALS patients. If approved, the drug could be marketed to patients.
Besides focusing on the trial, Feldman said shell have more time to do a deep dive into why people with diabetes get neurological complications.
She also was elected to the National Academy of Medicine and plans to use her position to help others understand the value of philanthropy.
Philanthropy can be a game changer Al Taubman showed us that and one of my goals is to help other clinician scientists throughout the country understand the process, she said.
Feldman said shes much closer to a cure for ALS, but were not there yet. For now, she wants to press the pause button on the disease.
When the patient comes to me, I want to be able to offer them a therapy so they will stay right where they are the first day I see them, she said. I dont think that stem cells will necessarily make people go back to where they are before they got ill, but if we can stop the disease in its tracks, my patients will be happy and I will be happy.
Feldman was named a 2011 Detroit News Michiganian of the Year. Shes served as the president of the American Neurological Association, the third woman to hold the position in 130 years, and was named one of Americas Top Doctors in 2016.
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Eva Feldman steps down as Taubman Institute director - The Detroit News
Banks targets embryonic stem cell research funding – Fort Wayne Journal Gazette
By JoanneRUSSELL25
Rep. Jim Banks, R-3rd, introduced legislation Thursday that would prevent the use of federal funds for stem cell research involving human embryos.
Banks' bill would direct the U.S. Department of Health and Human Services to give priority to medical research with the greatest potential for near-term clinical benefit in human patients and that does not use stem cells from destroyed, discarded or created embryos.
Scientists say embryonic stem cells show potential for transforming into other cells that might repair tissue damaged by disease or injury. Human embryonic stem cells used in research come from donated, unused fertilized eggs developed for in vitro fertilization procedures.
Adult blood stem cells are used to treat leukemia, and adult neural stem cells have been tested for brain disorders and spinal cord injuries.
This bipartisan bill prioritizes stem cell research that has a real impact on patients suffering right now while ensuring that research is conducted ethically without destroying human embryos, Banks, a freshman lawmaker from Columbia City, said in a statement.
Rep. Dan Lipinski, D-Ill., co-sponsored Banks' bill, which is called the Patients First Act of 2017.
The Dickey-Wicker Amendment of 1996 prohibited HHS from funding research using created or destroyed human embryos. But a federal court ruled in 2011 that Dickey-Wicker was ambiguous and did not ban research using stem cells from in vitro fertilization.
The Alliance for Regenerative Medicine, a coalition of medical companies, research institutions and patient advocacy groups that support embryonic stem cell research, had little to say Thursday about Banks' legislation.
As an organization representing the broader global regenerative medicine sector, our position is that we are in favor of government funds supporting the best science in an effort to speed safe and efficacious products to patients in need, Lyndsey Scull, senior communications director for ARM, said in an email.
Scull said ARM would monitor Banks' bill in the legislative process.
Banks' proposal states it would promote the derivation of pluripotent stem cell lines without the creation of human embryos for research purposes and without the destruction or discarding of, or risk of injury to, a human embryo.
The National Institutes of Health defines pluripotent stem cells as those that can give rise to any type of cell in the body except those needed to support and develop a fetus in the womb. They come from embryos and fetal tissue, although induced pluripotent stem cells are genetically reprogrammed cells taken from adult tissues.
In May, Banks led a letter signed by 40 other Republican House members that asked President Donald Trump to replace Dr. Francis Collins as the director of the NIH because of Collins' support for human embryonic stem cell research. Trump announced last week that he is retaining Collins, a geneticist nominated for NIH chief by President Barack Obama and confirmed by unanimous consent by the Senate in 2009.
The NIH is an HHS agency.
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Banks targets embryonic stem cell research funding - Fort Wayne Journal Gazette
Stem Cell Therapy Offers Hope for Multiple Sclerosis Remission – Healthline
By JoanneRUSSELL25
By combining an experimental stem cell treatment with a nanoparticle delivery system, researchers may eventually stop MS and other autoimmune diseases.
An innovative stem cell therapy could change how we treat multiple sclerosis (MS), but are we any closer to a cure?
The work of Dr. Su Metcalfe, founder and chief scientific officer of the biotech company LIFNano, appears to be breathing new life into that hope.
Metcalfe and her team developed a way to fight MS by using the bodys own natural mechanisms but it hasnt been tested in humans yet.
MS is an inflammatory and neurodegenerative autoimmune disease that can result in an array of neurological symptoms including fatigue, muscle spasms, speech problems, and numbness. It is caused by the immune system attacking myelin, the insulating coating that runs along the outside of nerve cells. The result is damage to the brain and central nervous system.
The disease currently affects roughly 2.5 million people worldwide. About 200 new cases are diagnosed each week in the United States.
LIFNano uses a new treatment based on LIF a stem cell protein that forms naturally in the body to signal and regulate the immune systems response to myelin.
LIF, in addition to regulating and protecting us against attack, also plays a major role in keeping the brain and spinal cord healthy, Metcalfe recently told Cambridge News.
In fact it plays a major role in tissue repair generally, turning on stem cells that are naturally occurring in the body, making it a natural regenerative medicine, but also plays a big part in repairing the brain when its been damaged, she said.
Metcalfe has spent years studying LIF, but only recently realized its potential for treatment likening it to an on/off switch for the immune system.
However, once she discovered its potential, there were almost immediate problems in its application. One of the earliest was how quickly LIF breaks down once it is administered into the body.
If you try just to inject it into a patient, it dissipates or disappears in about 20 minutes, Olivier Jarry, CEO of LIFNano, told Healthline.
That makes it unusable in a clinic. You would have to have some kind of pump and inject it continually.
A breakthrough came for Metcalfe when she took findings from her studies of LIF and applied them to nanotechnology. The treatment she is now developing relies on nanospheres derived from a well-established medical polymer known as PLGA, which is already used in materials like stitches. And because it is biodegradable, it can be left to dissolve inside the body.
Storing LIF inside these PLGA nanospheres before administering them into the bloodstream allows for a sustained dose over the course of several days.
The process differs significantly from the current drugs used to treat MS. These treatments most often fall under the category of drugs known as immunosuppressors, which inhibit the bodys overall immune system response.
LIF is theoretically much more precise than immunosuppressors, and should keep the immune system functioning against harmful infections and disease.
Were not using any drugs, said Metcalfe. Were simply switching on the bodys own systems of self-tolerance and repair. There arent any side effects because all were doing is tipping the balance. Autoimmunity happens when that balance has gone awry slightly, and we simply reset that.
The team cautions that LIF therapy is still several years away.
While some outlets have run wild with Metcalfes research, announcing that a cure for MS is right around the corner, those headlines are speculative.
Some MS advocacy groups have even made public statements calling coverage of her work premature and irresponsible.
Jarry told Healthline that LIFNano is expecting to enter FDA phase I trials in 2020. This would be the first time that it is used in human subjects. But even if the treatment proves to be safe and effective, the soonest it could be on the market is 2023, he estimated.
The main focus of LIF therapy is now on MS. But it has potential for treating other autoimmune diseases including psoriasis and lupus.
We are optimistic in the sense that we may provide a long-term remission for patients with MS, said Jarry.
Is it a cure? Wed love at some point to use the term cure, but we are very cautious.
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Stem Cell Therapy Offers Hope for Multiple Sclerosis Remission - Healthline
Rabbi has warning after concluding reviving dead ‘clearly possible’ – WND.com
By Sykes24Tracey
El Grecos Resurrection
A rabbi has issued a warning after concluding that raising the dead is clearly possible.
Its not for man to do.
The comments come from Rabbi Moshe Avraham Halperin and were reported by Breaking Israel News.
If they are able to revive a person from total brain death, it will be considered techiyat hamaytim [resurrection], he told BIN. Torah laws puts limits on man, forbidding him from some areas which are strictly divine. Reviving the dead is one of them.
Further, another rabbi said such experiments never will succeed.
The Rambam states that we must believe that the resurrection of the dead will happen when it is Gods will for it to take place and at no other time, Rabbi Yosef Berger, of King Davids Tom on Mount Zion in Jerusalem, told the news agency.
Transhumanism: Recreating humanity reveals the secret ways scientists are using technologyto pursue immortality, omniscience and ultimate power. Now available in the WND Superstore.
He was citing the 13 Principles of Faith set down by Rabbi Moses ben Maimon, whose 12th century work established him as a Torah authority and gave him the acronym Rambam.
He stated, I believe by complete faith that there will be a resurrection of the dead at the time that will be pleasing before the Creator.
That, Berger suggested, means, Not only does this effort by scientists go against this principle of faith, but we know that true resurrection can only happen by Divine will. Resurrection of the dead is described in depth, and it is proof of Gods rule over the physical world. But it is also stated that before the Messiah, there is no return from the grave.
The comments come on the heels of plans by a biomedical company, Bioquark, a startup based in Pennsylvania, to experiment with stem cells in an attempt to revive brain-dead patients.
BIN reported the company said it would launch experiments before the end of the year on such patients.
The trial will involve a multi-pronged approach, involving injecting stem cells and peptides into the spinal cords, electrical nerve stimulation, and laser therapy. The researchers hope this will grow new neurons and spur them to connect to each other, bringing the brain back to life, BIN reported.
The story pointed out that Bioquark reportedly tried such an experiment in India in 2016 but it was not with the approval of the nations drug regulators.
Amar Jesani, editor of the Indian Journal of Medical Ethics in Mumbai, cautioned that even partial success would traumatize families that had come to terms with a situation they believed irreversible. In point of fact, no families permitted their loved ones to be part of the experiment, BIN reported.
Halperin said, This is like using genetics to create a new form of life. There are realms that are strictly divine. Resurrection of the dead is clearly possible. It is definitely going to happen after the Messiah, but it restricted to God.
It was reported about a year ago that Bioquark and another company were embarking on the Reanima project, using a new drug formula involving stem cells.
Their plan was to use neurons, proteins, peptides and more that would create a microenvironment in which the stems cells can mature.
The report said, Inspired by organisms like salamanders that can regrow severed or damaged tails, Bioquark researchers have been developing regenerative treatments for a host of uses, from cancer to spinal cord injuries.
WND had reported when the company was given the green light on the visionary project.
The company said then it was capable of creating dynamics in mature tissues that are normally only seen during human fetal development, as well as during limb and organ regeneration in organisms like amphibians.
Christian author and filmmaker Tom Horn, at that time, said scientists are redefining what it means to be human, with the goal of transcending humanity.
Right here in North Carolina at your university, they have what is called a transgenic lab, which means they have mice that have human genetic material, for testing to see if the human parts in that animal are responding, he told TV host Sid Roth in a recent interview.
Using a gene-editing technique, one university lab cured cancer in a group of rats, but the unintended consequences were that the rats started aging very quickly and died at half-life, and nobody knows why that happened, Horn said. There is a danger in playing God because youre not God and you dont know.
Transhumanism: Recreating humanity reveals the secret ways scientists are using technologyto pursue immortality, omniscience and ultimate power. Now available in the WND Superstore.
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Rabbi has warning after concluding reviving dead 'clearly possible' - WND.com
Eva Feldman: Stepping down, not stopping – Crain’s Detroit Business
By Sykes24Tracey
Eva Feldman is stepping down as director of the A. Alfred Taubman Medical Research Institute at the University of Michigan, but at the age of 65, she has no intention of retiring.
She was the founding director of the institute created with tens of millions of dollars from mall mogul A. Alfred Taubman to fund research into intractable diseases like Alzheimer's, ALS and diabetes and ran it for 10 years. It is time, she says, for her next, but not necessarily last, 10-year plan.
"I birthed a baby and it's grown to be 10. It's a tweener, now, and ready for someone else to take it to adolescence," she told Crain's, following a meeting she had with one of the finalists of a national search to replace her.
Feldman said she expects a replacement to be named by the end of June.
"We started with four people, and now we have more than 200 investigators," she said. "We're strongly established, very robust. We have the best scientist-clinicians at the university. It's time for the next director to take over. There are other things I want to do, now. This takes up a lot of time, and I want to spend more time on my own research."
Feldman will continue to run the ALS Clinic at UM. Each Tuesday, she and a small staff diagnose and treat patients with amytrophic lateral sclerosis, sometimes known as Lou Gehrig's disease. The Russell N. DeJong Professor of Neurology at the U-M Medical School, she will also continue to run her own 30-scientist laboratory, the Program for Neurology Research & Discovery.
She said her new 10-year plan has three major goals.
This would be the last trial before the procedure is approved.
As soon as Feldman was done enumerating those goals, she enumerated two more.
She wants to help get more women into research positions at major universities. "More women than men graduate from the medical school at Michigan, but there are just a blip of women who are full professors," she said.
Last Wednesday, she and the Taubman Institute hosted a symposium called "Strategies to Empower Women to Achieve Academic Success," with keynote speakers on gender equality in academic medicine and a panel discussion on how to use negotiation and networking skills for career advancement.
And she wants to raise the $5 million it will take to fund the large-animal studies she needs to do before she can launch FDA human trials using embryonic stem cells to treat Alzheimer's. She said stem-cell trials with mice with dementia have been promising.
Researchers injected two groups of Alzheimer's mice, one group with a saline solution to serve as a control, the other with stem cells. Both of those groups and a group of healthy mice were then put through three tests of cognition, including one that required finding a platform hidden in a pool of water.
Previously, both sets of Alzheimer's mice flunked the cognition tests. After the stem-cell injections, the Alzheimer's mice injected with cells performed the tests as well as healthy mice. The Alzheimer's mice injected with saline solution flunked the tests.
"I am persistent. I will get that funded," she said.
Feldman took a sabbatical this spring to go to Australia, where she helped the Royal Children's Hospital in Melbourne set up a neuropathy-screening program for children with diabetes, which dovetails with one of her clinical trials treating neuropathy in children. And in September she heads to Chennai, India, to set up a similar program at a hospital there.
Neuropathy is a painful condition resulting from the nerve damage that diabetes can cause.
Feldman has published more than 350 peer-reviewed articles, 60 book chapters and three books and has had continuous funding from the National Institutes of Health for more than 20 years, including 12 current grants totaling about $5.5 million.
She is past president of the Peripheral Nerve Society and served as president of the American Neurological Association from 2011 to 2013, the group's third woman president in 130 years. Last June, she was named among the 100 most influential women in Michigan by Crain's.
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Eva Feldman: Stepping down, not stopping - Crain's Detroit Business
Patients volunteer for pioneering treatment by Cambridge scientist on verge of curing multiple sclerosis – Cambridge News
By Sykes24Tracey
Scores of people living with multiple sclerosis have put themselves forward for clinical trials as part of a Cambridge scientists bid to develop a treatment to cure the disease.
MS, an auto-immune condition which affects 2.3 million people around the world, attacks cells in the brain and the spinal cord, causing an array of physical and mental side effects including blindness and muscle weakness.
At the moment theres no cure, but Cambridge scientist Su Metcalfe and her company, LIFNano, hope to change that.
On Sunday the News told how Su has married one of the bodys cleverest functions with some cutting-edge technology. The natural side of the equation is provided by a stem cell particle called a LIF.
Su said: I discovered a small binary switch, controlled by a LIF, which regulates inside the immune cell itself. LIF is able to control the cell to ensure it doesnt attack your own body but then releases the attack when needed.
That LIF, in addition to regulating and protecting us against attack, also plays a major role in keeping the brain and spinal cord healthy. In fact it plays a major role in tissue repair generally, turning on stem cells that are naturally occurring in the body, making it a natural regenerative medicine, but also plays a big part in repairing the brain when its been damaged.
So I thought, this is fantastic. We can treat auto-immune disease, and weve got something to treat MS, which attacks both the brain and the spinal cord. So you have a double whammy that can stop and reverse the auto-immunity, and also repair the damage caused in the brain.
Sus company LIFNano has already attracted two major funding awards, from drug firm Merck and the Governments Innovate UK agency.
The company hopes to attract more investment, with the aim of starting clinical trials in 2020.
Off the back of the article regarding Sus work, people commented in their droves offering to trial Dr Metcalfes treatment, which is being touted as a possible cure.
Many also shared their experiences of living with MS.
James Stokes, 52 from Reading, was just one of many to comment on our story.
He told the News : I was diagnosed with MS on my 18th birthday of all days, but I wasnt told then that it was MS.
I had a big attack and was put in hospital; I lost my sight and couldnt speak or walk.
Even then I still wasnt officially told it was MS until a few years later.
About 10 years ago, things started to go downhill and I found myself basically housebound; I can still walk but not a great distance.
I use a walking stick, and have to use a wheelchair if Im going long distances.
MS has robbed me of my life, but when I saw this article there was a glimmer of hope.
Id love to know more about it [the process] and would be willing to take part in clinical trials.
If they could reverse my MS that would mean everything. At the moment, I feel as if Ive been given a life sentence but with no parole.
If a cure for MS is found, I could get a new lease of life and a lot more hope.
Many others also commented on the story regarding Sus work.
Debbie Brown wrote: This gives me some hope that things will improve in my lifetime. Diagnosed with RRMS in 2000. Very interested in registering for a clinical trial.
Amy Keighley said: My father was diagnosed with MS in 1996. He has been on several medications and is currently being treated by Dr Tornatori at Georgetown University Medical Center.
He fights every day and lives life to the fullest. He and my mother have been together since 1950. They were 15 years old. They are both retired from operating a jewellery store together for 23 years.
They try to travel every month and my dad's spirits remain high. I would like to ask that my dad be considered for the clinical trial.
Asterias Biotherapeutics to Present New 9-Month Efficacy Data from AST-OPC1 SCiStar Study at the International … – PR Newswire (press release)
By Sykes24Tracey
Workshop Details
Title: Clinical Development of hESC-derived Oligodendrocyte Progenitors for the Treatment of Spinal Cord injury Date: Tuesday, June 13, 2017 Time: 10:45 - 11:10 AM Room: 205ABC
Poster Presentation Details
Title: Safety and Efficacy of Human Embryonic Stem Cell-Derived Oligodendrocyte Progenitor Cells (AST-OPC1) in Patients with Subacute Cervical Spinal Cord Injury Poster Session III-ODD Poster Board F-1225 Date: Friday, June 16, 2017 Time: 6:00 - 7:00 PM
About the SCiStar Trial
The SCiStar trial is an open-label, single-arm trial testing three sequential escalating doses of AST-OPC1 administered at up to 20 million AST-OPC1 cells in as many as 35 patients with sub-acute, C-5 to C-7, motor complete (AIS-A or AIS-B) cervical SCI. These individuals have essentially lost all movement below their injury site and experience severe paralysis of the upper and lower limbs. AIS-A patients have lost all motor and sensory function below their injury site, while AIS-B patients have lost all motor function but may retain some minimal sensory function below their injury site. AST-OPC1 is being administered 14 to 30 days post-injury. Patients will be followed by neurological exams and imaging procedures to assess the safety and activity of the product.
The study is being conducted at six centers in the U.S. and the company plans to increase this to up to 12 sites to accommodate the expanded patient enrollment. Clinical sites involved in the study include the Medical College of Wisconsin in Milwaukee, Shepherd Medical Center in Atlanta, University of Southern California (USC) jointly with Rancho Los Amigos National Rehabilitation Center in Los Angeles, Indiana University, Rush University Medical Center in Chicago and Santa Clara Valley Medical Center in San Jose jointly with Stanford University.
Asterias has received a Strategic Partnerships Award grant from the California Institute for Regenerative Medicine, which provides $14.3 million of non-dilutive funding for the Phase 1/2a clinical trial and other product development activities for AST-OPC1.
Additional information on the Phase 1/2a trial, including trial sites, can be found at http://www.clinicaltrials.gov, using Identifier NCT02302157, and at the SCiStar Study Website (www.SCiStar-study.com).
About AST-OPC1
AST-OPC1, an oligodendrocyte progenitor population derived from human embryonic stem cells, has been shown in animals and in vitro to have three potentially reparative functions that address the complex pathologies observed at the injury site of a spinal cord injury. These activities of AST-OPC1 include production of neurotrophic factors, stimulation of vascularization, and induction of remyelination of denuded axons, all of which are critical for survival, regrowth and conduction of nerve impulses through axons at the injury site. In preclinical animal testing, AST-OPC1 administration led to remyelination of axons, improved hindlimb and forelimb locomotor function, dramatic reductions in injury-related cavitation and significant preservation of myelinated axons traversing the injury site.
In a previous Phase 1 clinical trial, five patients with neurologically complete, thoracic spinal cord injury were administered two million AST-OPC1 cells at the spinal cord injury site 7-14 days post-injury. They also received low levels of immunosuppression for the next 60 days. Delivery of AST-OPC1 was successful in all five subjects with no serious adverse events associated with AST-OPC1. No evidence of rejection of AST-OPC1 was observed in detailed immune response monitoring of all patients. In four of the five patients, serial MRI scans indicated that reduced spinal cord cavitation may have occurred. Based on the results of this study, Asterias received clearance from FDA to progress testing of AST-OPC1 to patients with cervical spine injuries, which represents the first targeted population for registration trials.
About Asterias Biotherapeutics
Asterias Biotherapeutics, Inc. is a biotechnology company pioneering the field of regenerative medicine. The company's proprietary cell therapy programs are based on its pluripotent stem cell and immunotherapy platform technologies. Asterias is presently focused on advancing three clinical-stage programs which have the potential to address areas of very high unmet medical need in the fields of neurology and oncology. AST-OPC1 (oligodendrocyte progenitor cells) is currently in a Phase 1/2a dose escalation clinical trial in spinal cord injury. AST-VAC1 (antigen-presenting autologous dendritic cells) is undergoing continuing development by Asterias based on promising efficacy and safety data from a Phase 2 study in Acute Myeloid Leukemia (AML), with current efforts focused on streamlining and modernizing the manufacturing process. AST-VAC2 (antigen-presenting allogeneic dendritic cells) represents a second generation, allogeneic cancer immunotherapy. The company's research partner, Cancer Research UK, plans to begin a Phase 1/2a clinical trial of AST-VAC2 in non-small cell lung cancer in 2017. Additional information about Asterias can be found at http://www.asteriasbiotherapeutics.com.
FORWARD-LOOKING STATEMENTS
Statements pertaining to future financial and/or operating and/or clinical research results, future growth in research, technology, clinical development, and potential opportunities for Asterias, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the businesses of Asterias, particularly those mentioned in the cautionary statements found in Asterias' filings with the Securities and Exchange Commission. Asterias disclaims any intent or obligation to update these forward-looking statements.
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SOURCE Asterias Biotherapeutics, Inc.
http://www.asteriasbiotherapeutics.com
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Asterias Biotherapeutics to Present New 9-Month Efficacy Data from AST-OPC1 SCiStar Study at the International ... - PR Newswire (press release)
Origin of age-related muscle loss discovered – Bel Marra Health
By raymumme
Home General Health Origin of age-related muscle loss discovered
When were young, we feel like we can accomplish anything. We can go anywhere and do anything because we dont feel encumbered by our physical limitations. However, when we age, we start to lose this feeling of empowerment. Our once-impressive physical physique fades away, leaving us weak and defenseless.
However, new research into the reasons why we lose muscle cells as we age may lead to the development of new drugs that can slow the process of muscle decline.
It was previously thought that the driving factor behind age-related muscle decline was the loss of motor neuronsnerve cells in the spinal cord controlling muscle. Starting in our 30s, we begin to notice that activities that once came easily are now difficult.
When we reach our 70s and 80s, we become weak and frail, having to depend on others to lend us their strength on a daily basis. No amount of diet and exercise can prevent this inevitable result.
The pursuit of preserving our youth has been a goal for many researchers and scientists. Understand how and why the body ages over time is something worth pursuing in the hopes of improving lives.
Research using mouse models has found that muscle stem cells play a very important role in lifelong maintenance of muscle. This challenges the current, widely accepted theory of motor neurons.
Muscle stem cell pools were seen to reside in muscle tissue that responds to exercise and injury. These stem cells are directly involved in repair and growth of muscles. It is the dying off of these stem cells that is theorized to be the driving factor for muscle loss.
Mice that were genetically altered to prevent muscle stem cell loss maintained healthier muscles in old age.
Subsequently, no evidence was found linking motor neurons to age-related muscle loss.
I think weve shown a formal demonstration that even for aging sedentary individuals, your stem cells are doing something. They do play a role in the normal maintenance of your muscle throughout life, said study author Joe Chakkalakal, Ph.D., assistant professor of Orthopaedics in the Center for Musculoskeletal Research at the University of Rochester Medical Center.
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Origin of age-related muscle loss discovered - Bel Marra Health
Stem cell-based spinal cord therapy expanded to more patients – The San Diego Union-Tribune
By Sykes24Tracey
An experimental therapy to repair spinal cord injury with stem cell-derived tissue is progressing smoothly, according to a leader of that trial who spoke at a conference on stem cell therapy.
The Phase 1 safety trial is proceeding with no complications, said Dr. Joseph Ciacci, a University of California San Diego neurosurgeon. The trial is being conducted at the universitys Sanford Stem Cell Clinical Center. The conference was held last week at the Sanford Consortium for Regenerative Medicine in La Jolla.
With safety looking good, the green light has been given to treat more patients, Ciacci said. However, to produce effectiveness, more cells will need to be transplanted.
Four patients have been treated with neural stem cells, injected into the spinal cord. They had experienced complete loss of motor and sensor function below the injury. They had been injured between 1 and 2 years previously.
Moreover, the cells show signs of integrating with the surrounding tissue in animal studies, Ciacci said. If the preliminary evidence holds up, Ciacci and colleagues plan to submit a paper detailing the results.
Curing paralysis from spinal cord injury was a big selling point for those who successfully advocated Proposition 71, which authorized selling $6 billion in state bonds to establish and fund the California Institute for Regenerative Medicine, or CIRM. The institute got $3 billion, the remaining half is going for interest over the life of the bonds.
While CIRM has been under pressure to show results, doctors are taking great care to establish safety first in the spinal cord treatment, because of potential risks in the procedure.
We are now enrolling and recruiting for the second cohort, which is for chronic cervical spinal cord injuries, Ciacci said. They are medically classified as C5-C7 ASIA A Complete.
Chronic injuries need to have taken place more than 1 year before treatment. For this study, the injury must also be under two years old. The trial is being conducted at UCSD with Ciacci serving as the principal investigator.
For more information on the Phase I Chronic SCI study, contact Ciaccis research group at (619) 471-3698, nksidhu@ucsd.edu.
In addition, the researchers have been approved to start another spinal cord injury trial with a different set of cells. These oligodendrocyte progenitor cells, derived from embryonic stem cells, can turn into several different types of neural cells.
The trial, sponsored by Asterias, treats newly injured patients, between 14 and 30 days after injury.
For more information on the Asterias trial, contact the UCSD Alpha Stem Cell Clinic at 858-534-5932 alphastemcellclinic@ucsd.edu or visit http://www.scistar-study.com and j.mp/ucsdast.
Asterias acquired the technology from Geron, which had undertaken the work with a CIRM grant. Geron later canceled the work and refunded the money to CIRM. Asterias got funding from CIRM to continue the work.
The Asterias trial will use the same technique as used with the Chronic SCI trial, a technique which can improve safety, Ciacci said. The cells will be injected in a series of progressively larger amounts that may give evidence of the dose relates to effectiveness, although safety remains the main concern.
This cell line is cryopreserved, its sent to us as a single dose the day of surgery, Ciacci said. Were going to study different doses 2 million, 10 million, 20 million cells per injection. Its going to be a direct injection, just like what weve done before.
As in previous treatments, patients will also receive immune suppression to prevent rejection of the cells. Likewise, they will be monitored for many years after treatment.
Another trial coming to UCSD will test for efficacy in ALS, Ciacci said.
Ciacci said hes looking for qualified patients for these trials, and urged those in the audience to help find them.
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bradley.fikes@sduniontribune.com
(619) 293-1020
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Stem cell-based spinal cord therapy expanded to more patients - The San Diego Union-Tribune
Bioquark aims to bring brain-dead people back to life next year – Metro – Metro
By NEVAGiles23
The Sun | Bioquark aims to bring brain-dead people back to life next year - Metro Metro It sounds like the plot of a horror movie - but a new trial aims to regenerate the brains of brain-dead people, by injecting them with stem cells. A U.S.... Controversial Study Wants To Resurrect Brain-Dead People ... US Firm Tries 'Reawakening The Dead' With Stem Cells [VIDEO] Company working on a way to reverse death |
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Bioquark aims to bring brain-dead people back to life next year - Metro - Metro
Meet the Cambridge scientist on verge of curing Multiple Sclerosis – Cambridge News
By LizaAVILA
Dr Su Metcalfe is sitting quietly reading through some documents in the lobby of the Judge Business School when I arrive for our interview. It would be easy to walk right past her and not know you were in the presence of a woman who could be on the verge of curing multiple sclerosis.
MS, an auto-immune condition which affects 2.3 million people around the world, attacks cells in the brain and the spinal cord, causing an array of physical and mental side effects including blindness and muscle weakness. At the moment theres no cure, but Su and her company, LIFNano, hope to change that.
Some people get progressive MS, so go straight to the severe form of the disease, but the majority have a relapsing or remitting version, she says.
It can start from the age of 30, and theres no cure, so all you can do is suppress the immune response, but the drugs that do that have side effects, and you cant repair the brain. The cost of those drugs is very high, and in the UK there are a lot of people who dont get treated at all.
But now a solution could be in sight thanks to Su, who has married one of the bodys cleverest functions with some cutting-edge technology. The natural side of the equation is provided by a stem cell particle called a LIF.
Su was working at the universitys department of surgery when she made her big breakthrough: I was looking to see what controls the immune response and stops it auto-attacking us, she explains.
I discovered a small binary switch, controlled by a LIF, which regulates inside the immune cell itself. LIF is able to control the cell to ensure it doesnt attack your own body but then releases the attack when needed.
That LIF, in addition to regulating and protecting us against attack, also plays a major role in keeping the brain and spinal cord healthy. In fact it plays a major role in tissue repair generally, turning on stem cells that are naturally occurring in the body, making it a natural regenerative medicine, but also plays a big part in repairing the brain when its been damaged.
So I thought, this is fantastic. We can treat auto-immune disease, and weve got something to treat MS, which attacks both the brain and the spinal cord. So you have a double whammy that can stop and reverse the auto-immunity, and also repair the damage caused in the brain.
Presumably Su, who has been in Cambridge since she was an undergraduate but retains a soft accent from her native Yorkshire, was dancing a jig of delight around her lab at this point, but she soon hit a snag; the LIF could only survive outside the cell for 20 minutes before being broken down by the body, meaning there was not enough time to deploy it in a therapy. And this is where the technology, in the form of nano-particles, comes in.
They are made from the same material as soluble stitches, so theyre compatible with the body and they slowly dissolve, says Su.
We load the cargo of the LIF into those particles, which become the delivery device that slowly dissolve and deliver the LIF over five days. The nano-particle itself is a protective environment, and the enzymes that break it down cant access it. You can also decorate the surface of the particles with antibodies, so it becomes a homing device that can target specific parts of the brain, for example. So you get the right dose, in the right place, and at the right time.
The particles themselves were developed at Yale University, which is listed as co-inventor with Su on the IP. But LIFNano has the worldwide licence to deploy them, and Su believes we are on the verge of a step-change in medicine.
She says: Nano-medicine is a new era, and big pharma has already entered this space to deliver drugs while trying to avoid the side effects. The quantum leap is to actually go into biologics and tap into the natural pathways of the body.
Were not using any drugs, were simply switching on the bodys own systems of self-tolerance and repair. There arent any side effects because all were doing is tipping the balance. Auto-immunity happens when that balance has gone awry slightly, and we simply reset that. Once youve done that, it becomes self-sustaining and you dont have to keep giving therapy, because the body has its balance back.
LIFNano has already attracted two major funding awards, from drug firm Merck and the Governments Innovate UK agency. Su herself is something of a novice when it comes to business, but has recruited cannily in the form of chairman Florian Kemmerich and ceo Oliver Jarry, both experienced operators in the pharma sector. With the support of the Judge, the company hopes to attract more investment, with the aim of starting clinical trials in 2020.
The 2020 date is ambitious, but with the funding weve got and the funding were hoping to raise, it should be possible, says Su.
Weve got everything we need in place to make the nano-particles in a clinically compliant manner, its just a case of flicking the switch when we have the money. Were looking at VCs and big pharma, because they have a strong interest in this area. Were doing all our pre-clinical work concurrently while bringing in the major funds the company needs to go forward in its own right.
Immune cells have been a big part of Sus career, and as we talk, her passion for her subject is obvious. I wanted to understand something that was so simple on one level but also so complex, she says.
The immune cell is the only single cell in the body that is its own unity, so it functions alone. Its probably one the most powerful cells in the body because it can kill you, and if you havent got it you die because you havent got it.
And MS may just be the start for LIFNano.
MS is our key driver at the moment, but its going to be leading through to other major auto-immune disease areas, Su adds.
Psoriasis is high up on our list, and diabetes is another. Downstream there are all the dementias, because a LIF is a major health factor for the brain. So if we can get it into the brain we can start protecting against dementia.
Now that would be something.
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Meet the Cambridge scientist on verge of curing Multiple Sclerosis - Cambridge News
Stem Cell Research Facts – Adult Stem Cell Success
By Dr. Matthew Watson
"I Will Walk Again" The Laura Dominguez Story If there was ever a woman on a mission, its Laura Dominguez. Doctors once told her shed never walk again. And while shes not ready to run a marathon, shes already proving them wrong, with the best yet to come.
An oil spill on a San Antonio freeway is blamed for the car crash that sent Laura and her brother directly into a retaining wall one summer afternoon in 2001. Laura was just 16 years old at the time and the crash left her completely paralyzed from the neck down. Surgeons say she suffered whats known as a C6 vertebrae fracture that severely damaged her spinal cord.
I refused to accept their prognosis that I never would walk again and began searching for other options, says Laura. After stays in several hospitals for nearly a year, Laura and her mother relocated to San Diego, CA so that she could undergo extensive physical therapy. While in California, they met a family whose daughter was suffering from a similar spinal cord injury. They were also looking for other alternatives to deal with spinal cord injuries.
After extensive research and consultations with medical experts in the field of spinal cord injuries, they decided to explore a groundbreaking new surgical procedure using adult stem cells pioneered by Dr. Carlos Lima of Portugal.
The surgery involved the removal of tissue from the olfactory sinus area at the back of the nose--and transplanting it into the spinal cord at the injury site. Both procedures, the harvesting of the tissue and the transplant, were done at the same time. Laura was the tenth person in the world and the second American to have this procedure done and was featured in a special report by PBS called Miracle Cell. (Link to Miracle Cell (PBS) Episode)
Following the surgery she returned to California where she continued with the physical therapy regimen, then eventually returned home to San Antonio. Upon her return home, an MRI revealed her spinal cord was beginning to heal. Approximately 70% of the lesion now looked like normal spinal cord tissue. More importantly to Laura, she began to regain feeling in parts of her upper body and within six months of the surgery regained feeling down to her abdomen.
Improvements in sensory feelings have continued until the present time. She can feel down to her hips, and has regained feeling and some movement in her legs. Lauras upper body has gained more strength and balance and one of the most evident improvements has been her ability to stand and remain standing, using a walker, and with minimal assistance. When she stands she can contract her quadriceps and hamstring muscles.
Every week it seems Im able to do something new, something different that I hadnt done the week before, says Laura.
Now Lauras story is poised to take a new, potentially groundbreaking turn. In the Fall of 2009, she traveled again to Portugal where adult stem cells were extracted from her nose for culturing. As this story is written, she is preparing to fly back to Portugal where scar tissue at her injury site will be removed and her own adult stem cells injected in the area of her original wound.
The Laura Dominguez story is not complete. The next chapter may or may not yield the results she seeksbut no one can deny the determination and courage of Laura. For her part, she has one goal in mind: I will walk again.
We shall update this site and keep you informed on her progress.
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Stem Cell Research Facts - Adult Stem Cell Success