Early research published in Mayo Clinic Proceedings examines the first case at Mayo Clinic of stem cell therapy tested in humans for spinal cord injury. The case study found stem cell intervention, which took place after standard surgery, and physical and occupational therapy, restored some function in a patient with spinal cord injury. The report, "Celltop Clinical Trial: First Report From a Phase I Trial of Autologous Adipose-Derived Mesenchymal Stem Cells in the Treatment of Paralysis Due to Traumatic Spinal Cord Injury" is published in the Nov. 27, 2019 edition of Mayo Clinic Proceedings.

The research discusses the experience related to the first case in a phase I safety study of mesenchymal stem cell treatment for spinal cord injury. Mohamad Bydon, M.D., a Mayo Clinic neurologic surgeon and the lead author, cautions that each patient is different, so it's too early to consider stem cell therapies as a treatment or cure for paralysis from spinal cord injury. Dr. Bydon adds that much like early trials in general, the stem cell trials are going to show variable response rates.

"Whilein this case, the first subject was a superresponder, others may not respond inthe same manner. We do not yet understand all of the necessary biology neededto achieve neurological recovery in paralyzed individuals," says Dr.Bydon. "One of our objectives in this study and future studies is tobetter delineate who will be a responder and why patients respond differently."

The research

The research centers on a 53-year-old man who suffered a spinal cord injury in a surfing accident that left him paralyzed below the neck. The patient had immediate improvements with standard therapy, but plateaued at six months post-injury. Researchers enrolled the patient in the study at Mayo Clinic nine months after the accident and injected the patient with stem cells 11 months after injury. After the stem cell injection, the patient significantly improved motor and sensory function.

Thecase study focuses on feasibility, safety and dosing of stem cell therapy. Thestudy team derived mesenchymal stem cells from the patient's fat cells andinjected them into the lower back in a procedure known as lumbar puncture.

Dr.Bydon; Wenchun Qu,M.D., Ph.D.,a physical medicine and rehabilitation physician; and Allan Dietz,Ph.D.,a transfusion medicine physician, led the multidisciplinary research team atMayo Clinic.

"Severespinal cord injury is a devastating condition for which scientists andphysicians are trying to find a cure. For the first time, we are inspiring hopethat people may receive better recovery in their function and quality of life,"says Dr. Qu. "Mayo Clinic has been taking the lead in translating thefruits of decades of research and treating neurological conditions, among whichhave been very important clinical trials where we evaluate the safety,feasibility and efficacy of adult stem cells for severe spinal cord injuries."

"This work both demonstrates the ability of cells to initiate repair and capitalizes on more than 10 years of work in the Immune, Progenitor and Cell Therapeutics Lab at Mayo Clinic. While there is still much to learn about the amazing ability of cells to heal tissue, this trial is an important step in advancing cell-based therapies toward clinical practice," says Dr. Dietz.

Investigatorscollected cerebrospinal fluid to look for new biological markers that mightgive clues to healing. Biological markers are important because they can helpidentify the critical processes that lead to spinal cord injury at a cellularlevel and could lead to new regenerative therapies.

Furtherstudy is needed to understand the effectiveness of stem cell lumbar injectionsand why patients may respond differently.

Currently,there is no way to reverse the devastating life-changing effects of paralysisfrom spinal cord injuries. Today, the only treatment is supportive care, suchas surgery and physical and occupational therapy.

Dr.Bydon says his early findings give hope that new regenerative therapies are onthe horizon for spinal cord injuries.

"Thehope is that we will have novel treatments for spinal cord injuries in the comingyears that will be different from what we have today. These will be therapies thatdo not rely upon supportive care, but therapies that rely on science to createa regenerative process for the spinal cord," says Dr. Bydon.

This research was made possible by funding from Mayo Clinic Transform the Practice Initiative and Regenerative Medicine Minnesota with support from the Mayo Clinic Center for Regenerative Medicine and the Department of Laboratory Medicine and Pathology Immune, Progenitor and Cell Therapeutics lab. The Transform the Practice Initiative aims to foster multidisciplinary teams of clinicians and researchers who align discovery and translational science, create new capacities and achieve solutions that improve the practice and address the unmet needs of patients.

###

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Tags: #Mayo Clinic Proceedings, #Spinal cord injury research, #stem cell lumbar puncture, #stem cell research, Dr. Allan Dietz, Dr. Mohamad Bydon, Dr. Wenchun Qu, Mayo Clinic Center for Regenerative Medicine, Regenerative Medicine Minnesota, Research, spinal cord injury

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Mayo Clinic research is a step toward hope for spinal cord ...

Spinal Cord Stem Cells Comments Off on Mayo Clinic research is a step toward hope for spinal cord … | March 25th, 2020

President Donald Trump has repeatedly said that the US is working to develop a vaccine for Covid-19, the disease caused by the novel coronavirus, as quickly as possible. But one of his own administrations policies appears to be standing in the way of at least one scientist.

According to a report by the Washington Posts Amy Goldstein, Kim Hasenkrug, an immunologist at the National Institutes of Healths Rocky Mountain Laboratories in Montana, wants to test potential treatments for Covid-19 in mice with humanized lungs. But as the Post first reported, the work is being held up by officials at the Department of Health and Human Services due to a 2019 ban on NIH scientists using donated fetal tissue from abortions in their research.

While fetal tissue isnt typically used to develop actual therapies or treatments, it has one particularly key use for researchers: the ability to create mice with human tissue suitable for medical testing. Mice, generally, have similar immune systems to humans, making them particularly useful for early medical testing.

Humanized mice have been key to developing several important medical treatments for diseases like the Zika virus or HIV/AIDS, which was Hasenkrugs previous research focus. The calculation is simple. You cant test certain treatments without humanized mice, and you cant get humanized mice without fetal tissue.

There are, of course, many avenues of research using other kinds of tissue, but fetal cells can rapidly divide, grow, and adapt to new environments in ways that make them the gold standard for some disease research. And in other research areas, we dont yet know if there is anything that could substitute, R. Alta Charo, professor of law and bioethics at the University of Wisconsin at Madison, wrote in the New England Journal of Medicine in 2015.

And as the Posts Goldstein noted, scientists have already shown that humanized mice could make good test subjects for coronavirus treatments specifically:

Just months ago, before the new coronavirus began to infect people around the world, other U.S. scientists made two highly relevant discoveries. They found that specialized mice could be transplanted with human fetal tissue that develops into lungs the part of the body the new coronavirus invades. These humanized mice, they also found, could then be infected with coronaviruses to which ordinary mice are not susceptible closely related to the one that causes the new disease, Covid-19.

Outside researchers have offered the mice to Hasenkrug for coronavirus research. But so far, Hasenkrug and other government researchers havent been allowed to obtain the mice they need to perform testing, the Post reported, thanks to a June 2019 HHS directive banning fetal tissue research for those employed by the government.

Caitlin Oakley, a HHS spokesperson, told the Post that no decision has been made about Hasenkrugs request. A separate HHS spokesperson confirmed that in a statement to Vox.

The spokesperson also pointed to an HHS statement from last June detailing the administrations policy on fetal tissue research. Promoting the dignity of human life from conception to natural death is one of the very top priorities of President Trumps administration, reads the statement.

Hasenkrug, and the potentially millions of Americans who may benefit from his research, now find themselves caught in a deeply divisive political issue thats been years in the making.

The US government had funded fetal tissue research efforts since the 1950s and for nearly as long, anti-abortion activists have opposed the practice.

In the Trump era, they finally found an administration ready to listen.

In 2018, the US government spent $115 million on about 173 research projects utilizing fetal tissue, a third of which were devoted to developing therapies for HIV/AIDS.

Research using fetal tissue has led to the development of vaccines such as those for polio, rubella, and measles, the International Society for Stem Cell Research (ISSCR) said in a statement last September. Fetal tissue is still helping advance science, with research underway using cells from fetal tissue to evaluate conditions including Parkinsons disease, ALS, and spinal cord injury. Fetal tissue is also necessary for the development of potential treatments for Zika virus and HIV/AIDS.

But anti-abortion activists argue it incentivizes abortion providers to perform more abortions in order to procure more tissue they could sell to third-party companies, which then provide the tissue directly to researchers. Fetal tissue procurement has been heavily regulated since enactment of the NIH Revitalization Act of 1993, which states that profits cannot be made in the transfer or acceptance of fetal tissue for research purposes.

That hasnt stopped anti-abortion activists from continuing to call into question the ethics of abortion providers or procurement companies. In 2000, the anti-abortion rights group Life Dynamics seemingly began the practice of releasing false or deceptively edited videos targeting the fetal tissue sales process. The main source in their videos was found to be not credible.

The George W. Bush administration did not take action against fetal tissue research, instead enacting restrictions on stem cell research derived from embryos in an August 2001 executive order. Those restrictions were later rolled back by an executive order from President Barack Obama in 2009.

More recently, the anti-abortion rights group Center for Medical Progress, run by activist David Daleiden, infamously released heavily edited videos appearing to show a Planned Parenthood employee negotiating prices for fetal tissue, and CMP accused the abortion care provider of illegally profiting from sales.

The videos caught the attention of Republican lawmakers. Investigations by the House Energy and Commerce, House Judiciary, and Oversight and Government Reform committees found no wrongdoing. Further investigations into Planned Parenthood and fetal tissue transfer proceeded with the creation of the Select Investigative Panel on Infant Lives in October 2015, chaired by Rep. Marsha Blackburn (R-TN), leading to $1.59 million in spending and a 471-page final report making numerable anti-abortion recommendations.

Among those requests was a call for the government to ban fetal tissue research by government scientists, which Barack Obamas administration, which favored the practice, ultimately ignored.

Democrats on the committee released their own report, disputing the conclusions of their Republican colleagues. At the end of their crusade, the conclusion was undeniable: There was no wrongdoing on behalf of fetal tissue researchers, including Advanced Bioscience Resources, or anyone else in the fetal tissue research space, said Rep. Jan Schakowksy (D-IL), who served as the ranking Democrat on the select committee, in a statement to Rewire.News in October 2018.

Anti-abortion activists saw an opportunity to advance their agenda on fetal tissue research when President Donald Trump won election in 2016, but it took a conservative media freakout in 2018 to enact new restrictions.

Over the summer of 2018, conservative media focused on several transactions by Advanced Bioscience Resources, a company that procured fetal tissue from abortion providers and shipped it to researchers for use. ABR was also one of the subjects of the 2015 select committee investigation.

HHS decided to cancel the governments contract with ABR in late September 2018 and began a review of the agencys rules and processes for procuring fetal tissue for research. That review concluded last summer, with HHS announcing in June that it would ban any fetal tissue studies by in-house NIH scientists, like Hasenkrug. It also introduced strict paperwork requirements for any outside scientists conducting research funded by the government.

The decision came as welcome news to anti-abortion activists. The language is trying to hold an ethical standard for the research proposals and the research that might be done. The policy is not just about science. Its also about ethics, David Prentice, vice president and research director at the anti-abortion Charlotte Lozier Institute, told Science magazine last July.

For his part, Hasenkrug has reportedly asked the Trump administration several times for permission to begin working with UNCs humanized mice for a coronavirus cure, but is still waiting on permission. Per the Post:

On Feb. 19, two people said, Hasenkrug wrote to a senior NIH official, asking for permission to use those mice and run experiments related to covid-19. He eventually was told that his request had been passed on to senior HHS officials.

Since then, he has written repeatedly to NIH, laying out in greater detail the experiments he wants to undertake and why several alternatives to the fetal tissue-implanted mice would not be as useful. In one appeal to NIH, Hasenkrug wrote that the mice he was offered are more than a year old and have a relatively short time remaining to live, so they should be used quickly, according to Kerry Lavender, a Canadian researcher familiar with the correspondence.

Hasenkrugs request has reportedly been forwarded to the White House Domestic Policy Council, which is chaired by Trump himself, but the government has not made a decision on the research as of yet.

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Coronavirus treatment research is delayed by Trumps ban on the use of fetal tissue - Vox.com

Spinal Cord Stem Cells Comments Off on Coronavirus treatment research is delayed by Trumps ban on the use of fetal tissue – Vox.com | March 20th, 2020

Edward Chang can't read your thoughts. Whenever the neuroscientist's lab at the University of California publishes a new piece of research, there's always a familiar refrain: that he's created "mind-reading technology" or can "read your thoughts". He's not alone, it's a phrase that follows much of the research into brain-computer interfaces and speech decoding.

And no wonder, when Elon Musk's startup Neuralink claims it will eventually enable "consensual telepathy" and Facebook one of the funders of Chang's lab said it wants to let people send messages by just thinking the words, rather than tapping them out on a phone, an example of a brain-computer interface (BCI).

But Chang isn't trying to read minds; he's decoding speech in people who otherwise can't speak. "We're not really talking about reading someone's thoughts," Chang says. "Every paper or project we've done has been focusing on understanding the basic science of how the brain controls our ability to speak and understand speech. But not what we're thinking, not inner thoughts." Such research would have significant ethical implications, but it's not really possible right now anyway and may never be.

Even decoding speech isn't easy. His most recent paper, in Nature last year, aimed to translate brain signals produced by speech into words and sentences read aloud by a machine; the aim is to help people with diseases such as amyotrophic lateral sclerosis (ALS) a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord. "The paper describes the ability to take brain activity in people who are speaking normally and use that to create speech synthesis it's not reading someone's thoughts," he says. "It's just reading the signals that are speaking."

The technology worked to an extent. Patients with electrodes embedded in their brains were read a question and spoke an answer. Chang's system could accurately decipher what they heard 76 per cent of the time and what they said 61 per cent of the time by looking at their motor cortex to see how the brain fired up to move their mouth and tongue. But there are caveats. The potential answers were limited to a selection, making the algorithm's job a bit easier. Plus, the patients were in hospital having brain scans for epilepsy, and could therefore speak normally; it's not clear how this translates to someone who can't speak at all.

"Our goal is to translate this technology to people who are paralysed," he says. "The big challenge is understanding somebody who's not speaking. How do you train an algorithm to do that?" It's one thing to train a model using someone you can ask to read out sentences; you scan their brain signals while they read out sentences. But how do you do that if someone can't speak?

Chang's lab is currently in the middle of a clinical trial attempting to address that "formidable challenge", but it's as yet unclear how speech signals change for those unable to speak, or if different areas of the brain need to be considered. "There are these fairly substantial issues that we have to address in terms of our scientific knowledge," he says.

Decoding such signals is challenging in part because of how little we understand about how our own brains work. And while systems can be more easily trained to move a cursor left or right, speech is complicated. "The main challenges are the huge vocabulary that characterise this task, the need of a very good signal quality achieved only by very invasive technologies and the lack of understanding on how speech is encoded in the brain," says David Valeriani of Harvard Medical School. "This latter aspect is a challenge across many BCI fields. We need to know how the brain works before being able to use it to control other technologies, such as a BCI."

And we simply don't have enough data, says Mariska van Steensel, assistant professor at UMC Utrecht. It's difficult to install brain implants, so it's not frequently done; Chang used epilepsy patients because they were already having implants to track their seizures. Sitting around waiting for a seizure to strike, a handful were willing to take part in his research out of boredom. "On these types of topics, the number of patients that are going to be implanted will stay low, because it is very difficult research and very time consuming," she says, noting that fewer than 30 people have been implanted with a BCI worldwide; her own work is based on two implants. "That is one of the reasons why progress is relatively slow," she added, suggesting a database of work could be brought together to help share information.

There's another reason this is difficult: our brains don't all respond the same. Van Steensel has two patients with implants, allowing them to make a mouse click with brain signals by thinking about moving their hands. In the first patient, with ALS, it worked perfectly. But it didn't in the second, a patient with a brain-stem stroke. "Her signals were different and less optimal for this to b e reliable," she says. "Even a single mouse click to get reliable in all situations is already difficult."

This work is different than that of startups such as NextMind and CTRL-Labs that use external, non-invasive headsets to read brain signals, but they lack the precision of an implant. "If you stay outside a concert hall, you will hear a very distorted version of what's playing inside this is one of the problems of non-invasive BCIs," says Ana Matran-Fernandez, artificial intelligence industry fellow at the University of Essex. "You will get an idea of the general tempo... of the piece that's being played, but you can't pinpoint specifically each of the instruments being played. This is the same with a BCI. At best, we will know which areas of the brain are the most active playing louder, if you will but we won't know why, and we don't necessarily know what that means for a specific person."

Still, tech industry efforts including Neuralink and Facebook aren't misplaced, says Chang, but they're addressing different problems. Those projects are looking at implant or headset technology, not the hard science that's required to make so-called mind reading possible. "I think it's important to have all of these things happening," he says. "My caveat is that's not the only part of making these things work. There's still fundamental knowledge of the brain that we need to have before any of this will work."

Until then, we won't be able to read speech, let alone inner thoughts. "Even if we were perfectly able to distinguish words someone tries to say from brain signals, this is not even close to mind reading or thought reading," van Steensel says. "We're only looking at the areas that are relevant for the motor aspects of speech production. We're not looking at thoughts I don't even think that's possible."

Edward Chang will be one of the speakers at WIRED Health in London on March 25, 2020. For more details, and to book your ticket, click here

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Why computers won't be reading your mind any time soon - Wired.co.uk

Spinal Cord Stem Cells Comments Off on Why computers won’t be reading your mind any time soon – Wired.co.uk | March 12th, 2020

Drug Target Review explores five of the latest research developments in the field of spinal cord injury (SCI) repair.

MRIs of Lumbar & Thoracic spine showing how a fracture of thoracic spine gets worse over time.

Researchers have shown that increasing energy supply to injured spinal cord neurons can promote axon regrowth and motor function restoration after a spinal cord injury (SCI).

We are the first to show that spinal cord injury results in an energy crisis that is intrinsically linked to the limited ability of damaged axons to regenerate, said Dr Zu-Hang Sheng, study co-senior author, senior principal investigator at the US National Institute of Neurological Disorders and Stroke (NINDS).

According to the team, energy levels are damaged because the mitochondria that produce adenosine triphosphate (ATP) for neurons are located in the axons. When damaged, the mitochondria are unable to produce ATP at the same level.

Nerve repair requires a significant amount of energy, said Dr Sheng. Our hypothesis is that damage to mitochondria following injury severely limits the available ATP and this energy crisis is what prevents the regrowth and repair of injured axons.

The scientists suggest that this is compounded by the anchoring of mitochondria in adult cells alongside the axons, so once damaged they are hard to replace.

Using a murine model, called a Syntaphilin knockout, where mitochondria are free to move along the axons, the researchers showed that when mitochondria are more mobile, mice have significantly more axon regrowth across the site of SCI compared to control animals. The paper also demonstrated that newly-grown axons made appropriate connections beyond the injury site, leading to functional recovery of motor tasks.

They hypothesised that increasing mitochondrial transport and thus the available energy to the injury site could enable repair of damaged nerve fibres.

When fed creatine, a compound that enhances the formation of ATP, both the control and knockout mice had increased axon regrowth following injury, compared to mice fed saline instead. More robust nerve regrowth was seen in the knockout mice that received creatine.

We were very encouraged by these results, said Dr Sheng. The regeneration that we see in our knockout mice is very significant and these findings support our hypothesis that an energy deficiency is holding back the ability of both central and peripheral nervous systems to repair after injury.

Dr Sheng highlighted that despite the promising results of the study published in Cell Metabolism, genetic manipulation was required for the best regrowth as creatine produced only modest regeneration. He concluded that further research is required to develop therapeutic compounds that are more effective in entering the nervous system and increasing energy production for the treatment of SCI.

Experiments exploring the role of immune and glial cells in wound healing and neural repair has revealed that Plexin-B2, an axon guidance protein, is essential for their organisation after SCI.

The researchers suggest their findings could aid in the development of therapies that target axon guidance pathways for treatment of SCI.

An artists impression of a macrophage.

The paper published in Nature Neuroscience reveals that Plexin-B2 on macrophages and microglia is essential for the process of corralling, where microglia and macrophages are mobilised and form a protective barrier around the site of SCI, separating healthy and necrotic tissue. In this study, researchers found that corralling begins early in the healing process and requires the ability of Plexin-B2 to steer immune cells away from colliding cells.

When they deleted Plexin-B2 from the microglia and macrophages in tissues, it led to tissue damage, inflammatory spillover and hindered axonal regeneration.

The lead investigator Dr Hongyan Jenny Zou, Professor of Neurosurgery and Neuroscience at the Icahn School of Medicine at Mount Sinai, US, said the results were quite unexpected.

She concluded that understanding the signalling pathways and interactions of glial cells with each other and the injury environment is fundamental to improving neural repair after a traumatic brain or spinal cord injury.

Another studyexploring the interactions of macrophages and microglia has revealed that in the central nervous system (CNS), microglia interfere with macrophages preventing them from moving out of damaged regions of the CNS.

We expected the macrophages would be present in the area of injury, but what surprised us was that microglia actually encapsulated those macrophages and surrounded them almost like police at a riot. It seemed like the microglia were preventing them from dispersing into areas they should not be, said Jason Plemel, a medical researcher at Canadas University of Alberta and a member of the Neuroscience and Mental Health Institute.

A microglial cell stained with Rio Hortegas silver carbonate method under the microscope.

Plemel said that more research is required to ascertain why this is happening, but they found that both the immune cells that protect the CNS, microglia and the immune cells of the peripheral immune system, macrophages, are present early after demyelination and microglia continue to accumulate at the expense of macrophages.

When we removed the microglia to understand what their role was, the macrophages entered into uninjured tissue. This suggests that when there is injury, the microglia interfere with the macrophages in our CNS and act as a barrier preventing their movement.

The scientists said that this observation was only possible because they were able to distinguish between microglia and macrophages, which has historically not been possible. Using this technique, they established than one type of microglia responded to demyelination. The results were published in Science Advances.

The indication of at least two different populations of microglia is an exciting confirmation for us, said Plemel. We are continuing to study these populations and hopefully, in time, we can learn what makes them unique in terms of function. The more we know, the closer we get to understanding what is going on (or wrong) when there is neurodegeneration or injury and being able to hypothesise treatment and prevention strategies.

Researchers suggest subpially-injecting neural precursor cells (NSCs) may reduce the risk of further injury associated with current spinal cell delivery techniques.

NSCs have the potential to differentiate into many neural cell types depending on the environment and have been the subject of investigation in both the field of SCI repair and neurodegenerative disease therapies.

subpially-injected cells are likely to accelerate and improve treatment potency in cell-replacement therapies for several spinal neurodegenerative disorders

However, the senior author of this study Dr Martin Marsala, professor in the Department of Anesthesiology at University of California (UC) San Diego School of Medicine, US, explained the current delivery techniques involve direct needle injection into the spinal parenchyma the primary cord of nerve fibres running through the vertebral column, so there is an inherent risk of (further) spinal tissue injury or intraparenchymal bleeding.

The novel technique Dr Marsala proposed in a paper published in Stem Cells Translational Medicine, is to inject these cells into the spinal subpial space an area between the pial membrane and the superficial layers of the spinal cord.

This injection technique allows the delivery of high cell numbers from a single injection, Dr Marsala explained. Cells with proliferative properties, such as glial progenitors, then migrate into the spinal parenchyma and populate over time in multiple spinal segments as well as the brain stem. Injected cells acquire the functional properties consistent with surrounding host cells.

The research collaborators suggest that subpially-injected cells are likely to accelerate and improve treatment potency in cell-replacement therapies for several spinal neurodegenerative disorders. This may include spinal traumatic injury, amyotrophic lateral sclerosis and multiple sclerosis, said study senior author Dr Joseph Ciacci, a neurosurgeon at UC San Diego Health.

The team now intend to move their experiments from rats to larger pre-clinical animal models, more anatomically similar to humans. The goal is to define the optimal cell dosing and timing of cell delivery after spinal injury, which is associated with the best treatment effect, concluded Dr Marsala.

Dr Mohamad Khazaei is the recipient of the STEM CELLS Translational Medicines (SCTM) Young Investigator Award for his work on SCI.

The award recognises advancements in the field of stem cells and regenerative medicine made by young researchers. The recipient is the principal author of an article published in SCTM that, over the course of a year, is deemed to have the most impact.

Dr Khazaeis work focuses on bringing cell-based strategies, such as NSC transplantation, into the therapeutic pipeline through generating and differentiating novel cell types using genetic and cell engineering approaches.

While we currently lack effective regenerative medicine treatment options for spinal cord injuries, Dr Khazaeis work to create a cell transplantation therapy utilising neural precursor cells is novel and provides a promising approach, said Dr Anthony Atala, Editor-in-Chief of SCTM and director of the Wake Forest Institute for Regenerative Medicine.

His winning paper details how Dr Khazaei and his team used neurons and oligodendrocytes to obtain better functional recovery after SCI.

Related topicsCell Regeneration, CNS, Disease research, Drug Delivery, Drug Discovery, Drug Targets, Neurons, Neurosciences, Regenerative Medicine, Research & Development, Therapeutics

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Exploring future spinal cord injury therapies - Drug Target Review

Spinal Cord Stem Cells Comments Off on Exploring future spinal cord injury therapies – Drug Target Review | March 12th, 2020

Adam Castillejo revealed himself to be the London Patient. Photo: Courtesy Adam Castillejo/Facebook

A London man who still has undetectable virus 30 months after stopping antiretroviral treatment is likely the second person ever cured of HIV, according to a report presented this week at the Conference on Retroviruses and Opportunistic Infections.

Two days before the conference was set to open in Boston, organizers decided to make the meeting virtual due to concerns about the coronavirus. Researchers gave their presentations via webcasts.

At last year's CROI, Dr. Ravindra Gupta of University College London reported that the so-called London Patient, who received a bone marrow transplant using stem cells from a donor with natural resistance to HIV, had no detectable virus in his blood plasma or T cells 18 months after stopping treatment.

At this week's meeting, Gupta said that an additional year of more extensive testing had found no functional HIV in the man's blood, lymph nodes, semen, gut tissue or cerebrospinal fluid.

"After 2.5 years off antiretrovirals and lack of evidence for any active virus, this almost certainly represents cure," Gupta told the Bay Area Reporter.

A day before Gupta's presentation, the New York Times revealed that the man, Adam Castillejo, 40, had decided to go public as the London Patient. Castillejo, who grew up in Venezuela, moved to London in 2002 and was diagnosed with HIV a year later. He is now leading a healthy and active life.

"My message to everyone out there living and coping with HIV is to not give up hope," Castillejo told the B.A.R. "I do hope that me going public will give some encouragement and empower people to keep breaking the stigma associated with HIV."

Resistant T cellsLike former San Francisco resident Timothy Ray Brown, known as the Berlin Patient the only other person known to be cured of HIV Castillejo underwent a bone marrow transplant to treat advanced cancer. According to the Times story, he spoke with Brown repeatedly before deciding to reveal his identity.

In both cases, doctors searched an international registry to find donors with double copies of an uncommon genetic mutation known as CCR5-delta-32, which makes T cells resistant to most types of HIV.

Brown received two stem cell transplants to treat leukemia in 2006, first undergoing strong chemotherapy and radiation to kill off his cancerous immune cells. He stopped antiretroviral therapy at the time of his first transplant, but his viral load did not rebound as expected. Over years of testing, researchers have found no functional virus anywhere in his body. Brown has now been free of HIV for more than 13 years.

Castillejo was diagnosed with lymphoma in 2011. After five years of grueling treatment, he underwent a bone marrow transplant in May 2016. But he received less aggressive chemotherapy than Brown and was able to stay on antiretroviral therapy.

The transplant led to complete remission of his lymphoma. Post-transplant tests showed that most of his T cells now lacked the CCR5 receptors HIV uses to enter the cells. In September 2017, with no evidence of viable HIV in his blood, he stopped his antiretrovirals in a closely monitored analytic treatment interruption.

When Castillejo was last tested on March 4, his plasma viral load remained undetectable using an ultrasensitive assay. Viral load was also undetectable in his semen and cerebrospinal fluid surrounding the brain and spinal cord. Biopsies showed no evidence of functional HIV in a lymph node or in his large or small intestine. Some bits of HIV genetic material were detected in long-lived memory T cells, but Gupta said these are probably "fossils" that cannot trigger active viral replication.

If this does prove to be a second cure, experts caution that the high-risk procedure will not be an option for people with HIV who do not need the treatment for cancer. But researchers are working on ways to mimic the same effect using gene therapy to delete CCR5 receptors from T cells or stem cells that give rise to all immune cells.

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Online Extra: London case appears to be second HIV cure - Bay Area Reporter, America's highest circulation LGBT newspaper

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10 things to know about stem cell therapy

New Delhi, March 3 (IANSlife) The usage of stem cells to cure or treat a disease or repair the injured tissue is defined as stem cell therapy. The best example of the stem cell treatment is seen in patients suffering from restoring the vision of the damaged eyes, grafting of the skin in severe burnt conditions. Stem cell treatments for brain or neural diseases like Parkinson''s and Alzheimer''s disease, multiple sclerosis, preventing heart strokes, curing diabetes, kidney disorders, autism, and spinal cord injuries are progressively making their way. Vipul Jain, CEO of Advancells and also a Serial entrepreneur, explains in detail the treatment, its uses, cost and effectiveness.

Q: What are stem cells?

Undifferentiated cells that are able to differentiate and transform into any type of cells of the body when and where needed. They have an enormous potential to repair, heal and regenerate. Stem cells come from blood, bone marrow, umbilical cord blood and adipose tissue.

Types of stem cell therapy

Autologous stem cell therapy: Patient receives stem cells from his/her own body

Allogeneic stem cell therapy: Patient receives the stem cells donated by another individual

Autologous stem cell therapy is better than allogeneic stem cell therapy as chances of mismatching are not there and they pose the minimum risk of immune rejection. Also, no side effects or adverse effects are seen as a person''s own blood cells are used. They start the healing process immediately in a natural way.

What is stem cell therapy?

The usage of stem cells to cure or treat a disease or repair the injured tissue is defined as stem cell therapy. Stem cells can be obtained from the bone marrow, adipose tissues etc. Due to their tremendous potential to prevent and to treat various health conditions and to repair the injured tissues global research investigation is continuously being done as to explore the maximum advantage of these cell lines.

The best example of the stem cell treatment is seen in patients suffering from restoring the vision of the damaged eyes, grafting of the skin in severe burnt conditions. Stem cell treatments for brain or neural diseases like Parkinson''s and Alzheimer''s disease, multiple sclerosis, preventing heart strokes, curing diabetes, kidney disorders, autism, and spinal cord injuries are progressively making their way.

What are the sources of stem cell?

Depending upon the disease, different stem cell source can be used in a specific condition. The procedure may involve the extraction of stem cells from adipose tissue-derived stem cells with the combination of PRP (Platelet-rich plasma) or can be obtained from bone marrow that can differentiate into progenitor cells that differentiate into various other tissues which can help in the therapy.

Procedure of stem cell therapy

The stem cells are isolated from the bone marrow or adipose tissues followed by their processing and enrichment under sterile conditions. These activated stem cells are placed back into the patient''s body at the target site for repairing the damaged tissue. It is necessary that the stem cells are injected in the specific area of injury as only then the desired results will be achieved.

Adipose stem cells are preferred over bone marrow stem cells as they are easy to isolate and contain a higher number of stem cells.

Stem cells injection

The stem cells injections are gaining much interest because it is devoid of the painful procedure, takes less time in comparison to a surgery, there are no host and recipient rejections as stem cells are harvested from the patient''s body itself and a targeted delivery system is available.

The stem cells obtained are processed in a sophisticated stem cell lab and after activation are inserted back into the host with the help of intravenous, intramuscular, intra-arterial, intradermal and intrathecal injections as per the requirement of the treatment process.

What is the use of anesthetics and why? Usually, local anesthetics are used during a stem cell procedure to numb the area but sometimes general anesthesia is also given while extracting the stem cells from bone marrow. But it is necessary to find out what anesthetic your doctor uses during orthopedic stem cell treatments.

A number of anesthetics have been found to kill the stem cells thus; the treatment''s end result will greatly depend on the use of anesthetics. Some anesthetics very well sync with the stem cell and hence, aid in the treatment.

How good are the processing techniques in the onsite labs?

Stem cells are to be extracted and processed in a clean room, under aseptic conditions maintaining a controlled environment. The doctor should explain the entire process and the number of viable stem cells infused into the patient during the process. Also, the precision of the injections to provide good quality of stem cells at the site of injury will help in better and faster recovery of the patient''s damaged area.

Duration and cost of the therapy

Cost of the treatment and its duration varies from one patient to another. The disease which needs to be cured, the severity, age factor, health condition, etc, define the duration of the therapy. One may respond during the treatment phase itself while the other may show results after a few sessions or weeks. Depending upon the disease diagnosed, the stem cells extracted, duration of the therapy, other adjuvants used in the process, the cost of the stem cell therapy can vary.

Follow-up visits

It is essential that after the stem cell therapy the patient should visit the stem cell doctor for recuperation therapies. The primary goals of such therapy is the prevention of secondary complications, analysis of recovery of motor, sensory and all the bodily functioning, psychological support/counseling for depression, mood swings or anxiety etc. and reintegration into the community.

There can be different sets of precautions which need to be followed at various steps for the recovery of the damaged tissues. The treatment and post treatment conditions may vary from person to person depending upon the disease and the severity.

Success rate of stem cell therapy

Stem cell therapy has shown results in treating serious ailments like leukemia, grafting tissues, autism, orthopedic conditions and skin problems etc. Stem Cell Therapy has been successfully used in the treatment of around 80 serious disorders.

Survival rates among patients who received stem cell treatment are significantly high, whether the cell donors are related or unrelated to them. With the ongoing research around the world, scientists are exploring new possibilities in which a number of life threatening diseases can be prevented and cured hence, the stem cells have proved to be promising in the near future as many aspects are yet to be revealed.

--IANS

pg/adr/

Disclaimer :- This story has not been edited by Outlook staff and is auto-generated from news agency feeds. Source: IANS

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10 things to know about stem cell therapy - Outlook India

Spinal Cord Stem Cells Comments Off on 10 things to know about stem cell therapy – Outlook India | March 4th, 2020

Pune, March 03, 2020 (GLOBE NEWSWIRE) -- The global Spinal Muscular Atrophy Treatment Market size is expected to reach USD 14.49 billion by 2026, exhibiting a CAGR of 28.9% during the forecast period. The rising prevalence of rare diseases around the world will fuel demand for SMA treatment in the forthcoming years, which in turn will aid the growth of the market. As per the National Policy for the treatment of rare diseases, globally, around 6000 to 8000 rare diseases are estimated to exist with new rare diseases reported on a regular basis. Furthermore, 80% of all the rare diseases are genetically originated and therefore impact children inexplicably. The survey also revealed that 50% of new cases are in children and are responsible for 35% of deaths before the age of 1 year, 10% between the ages of 1 and 5 years and 12% between 5 and 15 years. Nonetheless, "the growing initiatives by government authorities for pre-diagnosis will impact the Spinal Muscular Atrophy Treatment Market share positively during the forecast period", predicts our lead analysts at Fortune Business Insights.

For more information in the analysis of this report, visit: https://www.fortunebusinessinsights.com/industry-reports/spinal-muscular-atrophy-treatment-market-100576

According to the report, published by Fortune Business Insights, titled "Spinal Muscular Atrophy Treatment Market Size, Share and Global Trend By Product (Nusinersen and Onasemnogen Abeparvovec), By Disease Type (Type 1 SMA, Type 2 SMA and Others), By Distribution Channel (Hospital Pharmacies, Retail Pharmacies and Others), and Geography Forecast till 2026" the market size stood at USD 1.72 billion. The SMA Treatment Market report executes a PESTEL study and SWOT analysis to reveal the stability, restrictions, openings, and threats in the smart building market. Combined with the market analysis capabilities and knowledge integration with the relevant findings, the report has foretold the robust future growth of the SMA treatment market, and all articulated with geographical and merchandise segments. Moreover, it also shows different procedures and strategies, benefactors and dealers working in the market, explores components convincing market development, generation patterns, and following systems. Additionally, the figures and topics covered in this report are both all-inclusive and reliable for the readers.

Market Driver:

R&D Initiatives by Key Players to Spur Sales Opportunities

The surge in research and development activities for the improvement of therapies and treatment options by key players will aid the Spinal Muscular Atrophy Treatment Market growth during the forecast period. Various drug pipeline for advanced stages of clinical trials by major pharmaceutical companies will augment the healthy growth of the market. For instance, Genentech/Roche's pipeline candidate of Risdiplam, which recently received a priority review from the FDA and is expected to receive a decision on approval from the FDA by May 2020. Furthermore, the growing initiatives for pre-diagnosis and positive reimbursement policies will boost the Spinal Muscular Atrophy Treatment Market trends in the foreseeable future. Moreover, the growing awareness regarding pivotal treatment options will create new opportunities for the market.

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Market Restraint:

High Cost of Products to Impede Market Expansion

The cost-intensive products and high prices associated with the rare disease therapies will subsequently obstruct the growth of the market. For instance, spinraza is expected to cost US$ 750,000 for the first year and will be repriced at US$ 375,000 after that. Apart from that, Novartis rare gene therapy, Zolgensma will come at a price of US$ 2.1 million for a one-time treatment. The expensive cost of therapies will restrict the adoption of treatment for many patients, which in turn will act as a restraint for the Spinal Muscular Atrophy Treatment Market revenue.

Regional Insight:

Presence of Major Players to Influence Growth in North America

The market in North America stood at USD 854 million in 2018 and is likely to remain dominant during the forecast period. The growth in the region is attributed to the presence of prominent players in the region. The growing awareness regarding the prevalence of rare disease and pre-treatment initiatives will bolster accelerate the Spinal Muscular Atrophy Treatment Market growth in North America.

List of the Major Players Operating in the Global SMA Treatment Market Include:

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Spinal Muscular Atrophy Treatment Market to Exhibit a Spectacular CAGR of 28.9%; Growing Initiatives by Government Authorities for Pre-Diagnosis to...

Spinal Cord Stem Cells Comments Off on Spinal Muscular Atrophy Treatment Market to Exhibit a Spectacular CAGR of 28.9%; Growing Initiatives by Government Authorities for Pre-Diagnosis to… | March 4th, 2020

People sustain approximately 11,000 spinal cord injuries each year. Approximately 38 percent of these injuries are from motor vehicle accidents, with falls, gunshot wounds and similar forms of violence, sports activities, and medical or surgical complications being the other common causes. Regardless of the cause, any type of injury to the spinal cord can damage nerve and tissue cells and result in a loss of sensation or paralysis. One possible option for treatment for spinal cord injuries thats been gaining traction among Beverly Hills spine surgeons is the use of stem cells.

Stem cells are undifferentiated cells. They are useful because they can become specialized cells in the area where they are injected. These cells have the ability to become other types of cells and encourage the production of new, healthy cells. There are two common types of stem cells:

Normally, treatment for a spinal cord injury involves extensive physical therapy and rehab. However, recovery is usually limited because an injured spine cant heal due to the formation of scar tissue triggered by an inflammatory response that keeps healthy cells from reaching the damaged area.

Adult stem cells that come from either bone marrow or a donated umbilical cord from a healthy pregnancy are usually used for spinal injury treatments. With umbilical cord tissue, there is a more rigorous screening process to look for viruses and bacteria. Regardless of how they are collected, stem cells may help with spinal cord injuries by:

Along with a local anesthetic, stem cells used to treat spinal injuries are injected directly into the affected area, and they are placed into the spinal fluid to allow the undefined cells to reach the injured part of the spine. Patients usually receive multiple injections over the course of several weeks. Treatment is often coupled with:

Stem cells wont completely repair an injured spinal cord. However, there are several promising studies that suggest some patients do see noticeable improvements, such as the ability to feel light touch below the injured area. At one facility in India, eight out of ten patients with no motor or sensory function below the waist were able to walk for about an hour with the assistance of a walker eight months after receiving transplanted stem cells. Stem cell therapy is still in its infancy, but it does offer some hope for patients with spinal injuries who are looking for an alternative to minimally invasive spinal surgery. Beverly Hills residents should contact The Spine Institute at 310-828-7757 for more information.

According to an article on Beckers Spine Review, The Spine Institutes Dr. Hyun Bae has spent a significant amount of time researching stem cell repair for degenerative disc disease as well as how growth factors can treat spinal cord injuries. Dr. Bae was among the first spinal surgeons to utilize growth factor tissue engineering for intervertebral discs, and in 2010 he also chaired a course for the North American Spine Society that dealt with navigating research in spinal biologics.

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Stem Cells: A New Way to Potentially Treat Spinal Cord ...

Spinal Cord Stem Cells Comments Off on Stem Cells: A New Way to Potentially Treat Spinal Cord … | February 28th, 2020

Last summer, U.S. Army Lieutenant Colonel Jason Barnhill traveled to an undisclosed desert location in Africa with a ruggedized 3D printer and other basic supplies that could be used to biofabricate for field medical care, such as human mesenchymal stem/stromal cells (hMSCs). The aim was to discover how a 3D bioprinter could expedite healing and even replace damaged tissue for troops injured in combat.

Jason Barnhill with a 3D bioprinter that could replace damaged tissues for troops injured on the battlefield. (Image: Military Health System/West Point)

Barnhill, who is the life science program director of the United States Military Academy West Point Department of Chemistry and Life Sciences, is leading a project with a team of cadets working on experiments to advance bioprinting research in the field with an ultimate goal to develop technology for creating wound-healing biologics, bandages, and more for soldiers on-site or near the point-of-care. According to U.S. Army news, 26 first-class cadets at the United States Military Academy at West Point, in New York, are doing bioprinting research across seven different projects: two teams are working on biobandages for burn and field care; other two teams are working on how to bioengineer blood vessels to enable other bioprinted items that require a blood source, such as organs, to be viable; while one team is working on printing a viable meniscus, and another team is looking to print a liver.

A lot of this has to do with the bioink that we want to use, exactly what material were using as our printer ink, if you will, explained Class of 2020 cadet Allen Gong, a life science major conducting research for the meniscus project. Once we have that 3D model where we want it, then its just a matter of being able to stack the ink on top of each other properly.

Gong, along with his teammates, are researching how to use bioinks to create a meniscus that could be implanted into a soldiers injured knee, while other cadets are seeking to print a liver that could be used to test medicine and maybe one day eliminate the shortage of transplantable organs. This is not the first time we hear the U.S. Army is using bioprinting for regenerative medicine, after all, they often suffer from trauma, resulting in loss of limbs, injuries to the face and severe burns. Deployed soldiers confront the risks of battle on a daily basis. However, being able to have immediate access to specialized bioprinters created to solve catastrophic medical injuries could be the dream-scenario solution many have been waiting for.

In 2014, scientists at the Armed Forces Institute of Regenerative Medicine (AFIRM), established by the Department of Defense, were using 3D bioprinters extensively for skin repair research; but the Army is also actively developing artificial 3D printed hearts, blood vessels, and other organs in a quest to develop customizable and 3D printed medicine. Barnhills pilot program in 2019, conducted by the Uniformed Services University of the Health Sciences (USU) in collaboration with the U.S. Military Academy at West Point, has shown that a 3D printer capable of biofabrication could potentially change the way deployed warfighters receive care also. Under his direction, the 3D printer successfully fabricated a number of products, including a scalpel capable of immediate use and a hemostat (a surgical tool used to control bleeding during surgery and capable of gripping objects) while locking them into place to hold a tissue or other medical implements. The tools were made of a material that could be sterilized on-site, reducing the chance of infection during practical use.

Common combat injuries include second and third-degree burns, broken bones, shrapnel wounds, brain injuries, spinal cord injuries, nerve damage, paralysis, loss of sight and hearing, post-traumatic stress disorder (PTSD), and limb loss. Many of these injuries could be tackled with customizable, on-site bioprinting machines, but for now, the cadets on each of the teams are in the beginning stages of their research before starting the actual printing process. This stage includes reading the research already available in their area of focus and learning how to use the printers, and after spring break, they will have their first chance to start printing with cells. The teams focusing on biobandage, meniscus, and liver will try to print a tangible product by the end of the semester as part of the initial research.

Another cadet and life science major working on the meniscus project, Thatcher Shepard, described in the U.S. Army article that there are definitely some leaps before we can get to that point [of actually implanting what they print]. We have to make sure the body doesnt reject the new bioprinted meniscus and also the emplacement. There can be difficulties with that. Right now, were trying to just make a viable meniscus, then, well look into further research to be able to work on methods of actually placing it into the body.

They claim that the meniscus team is starting with magnetic resonance images (MRI) of knees and working to build a 3D model of a meniscus, which they will eventually be able to print. A great deal of the teams research will be figuring out how and when to implant those cells into the complex cellular structure they are printing.

Cadets at West Point Department of Chemistry and Life Sciences (Image: West Point)

According to Michael Deegan, another life science major and cadet working on one of the blood vessel projects, for now, it will involve a lot of research into what has already been done in the field and the questions that still need to be answered. He described the experience as kind of like putting the cart before the horse. Saying that youve printed it, great, but whats the point of printing it if its not going to survive inside your body? Being able to work on that fundamental step thats actually going to make these organs viable is what drew me and my teammates to be able to do this. Deegan and his colleagues will eventually decide on the scope and direction of their projects, knowing that their research will be key to allowing other areas of the field to move forward, since organs, such as livers and pancreases, have been printed, but so far, they can only be produced at the micro level because they have no blood flow.

While generating organs and blood vessels will be one of the great benefits of customized medicine in the future, the work behind the biobandage teams could have a direct use in the field during combat. The U.S. Army suggests that the goal is to be able to take cells from an injured soldier, specifically one who suffered burns and print a bandage with built-in biomaterial on it to jumpstart the healing process. Medical personnel could potentially be deployed with a 3D printer in their Forward Operating Base or it could be sent along in a column with a Humvee to enable bandages to be printed on-site.

Were researching how the body actually heals from burns, said Channah Mills, a life science major working on one of the biobandage projects. So, what are some things we can do to speed along that process? Introducing a bandage could kickstart that healing process. The faster you start healing, the less scarring and the more likely youre going to recover.

Being on the forefront of it and just seeing the potential in bioengineering, its pretty astounding, Gong said. But it has also been sobering just to see how much more complicated it is to 3D print biomaterials than plastic.

At the moment, the projects are building on existing research on printing sterile bandages and then adding a bioengineering element. The bandages would be printed with specialized skin and stem cells necessary for the healing process.

More than half of the cadets working on the bioprinting projects plan to continue on to medical school following their graduation from West Point. This research, which will be presented during the academys annual Projects Day on April 30, is a great starting point for the future army doctors, as they begin to understand and work on some of the more complex technologies that could become their allies in the future, helping them heal soldiers in the field.

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West Point: Bioprinting for Soldiers in the Battlefield - 3DPrint.com

Spinal Cord Stem Cells Comments Off on West Point: Bioprinting for Soldiers in the Battlefield – 3DPrint.com | February 28th, 2020

Neuroplast is a company based in Maastricht (the Netherlands) developing autologous stem cell therapies for patients suffering from neurodegenerative diseases such as spinal cord injury (SCI), amyotrophic lateral sclerosis (ALS) and traumatic brain injury.

Recently, the company has raised 4 million from Dutch-based Brightlands Venture Partners and LIOF and from an existing shareholder and informal investor Lumana Invest BV.

CEO Johannes de Munter said:

The financing and support of the investors will enable us to perform multicenter clinical trials in the Netherlands, Denmark, Germany, and Spain and bring the product to market.

This Dutch startup will use the fund to perform a phase II/III clinical trial with the aim of obtaining conditional market approval for the treatment of patients suffering from Spinal Cord Injury.

Founded by physician Hans de Munter and neurologist Erik Wolters in 2014, Neuroplast has expanded with Juliette van den Dolder, who was appointed as COO and management team member.

In the case of SCI, isolating, manufacturing, and reinserting patients own cells, very promising preclinical outcomes have resulted in an Orphan Drug Designation from European regulatory authorities, allowing a fast-track procedure for the clinical trials. These trials are expected to start in March 2020.

Marcel Kloosterman Director at Brightlands Venture Partners:

Neuroplast combines breakthrough science with a solid management team. In a sizable market characterised by major unmet need, successful treatment of (accident caused) paralysed patients would make life so much easier for them and their families while lowering the burden and costs for the society.

Yearly, 24,500 people in Europe and the USA are diagnosed with Spinal Cord Injury, usually caused by accident. Its worth mentioning that for Europe and the US, the medical cost associated with Spinal Cord Injury is over 13 bn per year.

CEO Johannes de Munter adds:

Neuroplast is becoming an ATMP player in the region and wants to contribute to our beautiful eco-system.

Main image credits:Neuroplast

Stay tuned toSilicon Canalsfor more European technology news

Originally posted here:
Dutch startup Neuroplast raises 4M for its stem cell-based technology to treat patients with Spinal Cord Injury - Silicon Canals

Spinal Cord Stem Cells Comments Off on Dutch startup Neuroplast raises 4M for its stem cell-based technology to treat patients with Spinal Cord Injury – Silicon Canals | February 20th, 2020

U.S. Department of Defense Researchers to Study Ability of siFi2 to Drive Axon Regeneration and Functional Recovery following Spinal Cord Injury

NEW YORK, Feb. 19, 2020 (GLOBE NEWSWIRE) -- MicroCures, a biopharmaceutical company developing novel therapeutics that harness the bodys innate regenerative mechanisms to accelerate tissue repair, today announced that it has entered into a material transfer agreement (MTA) with the Henry M. Jackson Foundation (HJF) for the Advancement of Military Medicine. Under terms of the agreement, United States Department of Defense researchers will conduct a preclinical study of siFi2, MicroCures lead product candidate, in animal models of spinal cord injury. siFi2, a small interfering RNA (siRNA) therapeutic that can be applied topically, is designed to enhance recovery after trauma.

Researchers, led by Kimberly Byrnes, Ph.D. of Uniformed Services University of the Health Sciences, will evaluate the potential of siFi2 treatment to drive axon regeneration and functional recovery in a rat model of spinal cord injury. As part of this study, multiple siFi2 formulations will be evaluated in order to assist in the identification of a lead formulation to be advanced into clinical development.

MicroCures technology is based on foundational scientific research at Albert Einstein College of Medicine regarding the fundamental role that cell movement plays as a driver of the bodys innate capacity to repair tissue, nerves, and organs. The company has shown that complex and dynamic networks of microtubules within cells crucially control cell migration, and that this cell movement can be reliably modulated to achieve a range of therapeutic benefits. Based on these findings, the company has established a first-of-its-kind proprietary platform to create siRNA-based therapeutics capable of precisely controlling the speed and direction of cell movement by selectively silencing microtubule regulatory proteins (MRPs).

The company has developed a broad pipeline of therapeutic programs with an initial focus in the area of tissue, nerve and organ repair. Unlike regenerative medicine approaches that rely upon engineered materials or systemic growth factor/stem cell therapeutics, MicroCures technology directs and enhances the bodys inherent healing processes through local, temporary modulation of cell motility. siFi2 is a topical siRNA-based treatment designed to silence the activity of Fidgetin-Like 2 (FL2), a fundamental MRP, within an area of wounded tissue or nerve. In doing so, the therapy temporarily triggers accelerated movement of cells essential for repair into an injury area. Importantly, based on its topical administration, siFi2 can be applied early in the treatment process as a supplement to current standard of care.

The U.S. Department of Defense continues to be a valued and trusted partner for MicroCures as we work to advance research of siFi2 with the goal of ultimately delivering transformative treatments to patients with significant unmet medical needs, said David Sharp, Ph.D., co-founder and chief science officer of MicroCures. With a focus in the area of spinal cord injury, this MTA further demonstrates the broad applicability of our technology platform to a range of therapeutic indications. We look forward to collaborating with Dr. Byrnes and her team at Uniformed Services University of the Health Sciences to continue the advancement of this promising program.

Previously conducted research in a rat model of spinal cord injury has demonstrated that treatment with siFi2 allowed axon growth to occur through the inhibitory barriers that typically appear and prevent healing at the site of injury. Conversely, study results failed to demonstrate similar axon growth through these inhibitory barriers for animals administered a siRNA control treatment. Additional preclinical findings have demonstrated functional improvement in rats with spinal cord injury following treatment with siFi2. This was evidenced by significantly improved hind limb locomotor function in siFi2-treated animals as compared to control subjects at Day 5 (p < 0.05) and Day 7 (p < 0.01).

About MicroCures

Story continues

MicroCures develops biopharmaceuticals that harness innate cellular mechanisms within the body to precisely control the rate and direction of cell migration, offering the potential to deliver powerful therapeutic benefits for a variety of large and underserved medical applications.

MicroCures has developed a broad pipeline of novel therapeutic programs with an initial focus in the area of tissue, nerve and organ repair. The companys lead therapeutic candidate, siFi2, targets excisional wound healing, a multi-billion dollar market inadequately served by current treatments. Additional applications for the companys cell migration accelerator technology include dermal burn repair, corneal burn repair, cavernous nerve repair/regeneration, spinal cord repair/regeneration, and cardiac tissue repair. Cell migration decelerator applications include combatting cancer metastases and fibrosis. The company protects its unique platform and proprietary therapeutic programs with a robust intellectual property portfolio including eight issued or allowed patents, as well as eight pending patent applications.

For more information please visit: http://www.microcures.com

Contact:

Vida Strategic Partners (On behalf of MicroCures)

Stephanie Diaz (investors)415-675-7401sdiaz@vidasp.com

Tim Brons (media)415-675-7402tbrons@vidasp.com

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MicroCures Announces Material Transfer Agreement with Henry M. Jackson Foundation for the Advancement of Military Medicine to Support Preclinical...

Spinal Cord Stem Cells Comments Off on MicroCures Announces Material Transfer Agreement with Henry M. Jackson Foundation for the Advancement of Military Medicine to Support Preclinical… | February 20th, 2020

Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs, announced today that updated results from a Phase I/IIa study of its lead product candidate, OpRegen, a retinal pigment epithelium (RPE) cell transplant therapy currently in development for the treatment of dry age-related macular degeneration (dry AMD), have been accepted for presentation at the 2020 Association for Research in Vision and Ophthalmology (ARVO) Meeting, which will be held May 3rd through May 7th, 2020 at the Baltimore Convention Center in Baltimore, MD. The abstract presentation, entitled, "Phase I/IIa Clinical Trial of Human Embryonic Stem Cell (hESC)-Derived Retinal Pigmented Epithelium (RPE, OpRegen) Transplantation in Advanced Dry Form Age-Related Macular Degeneration (AMD): Interim Results", will be presented as part of the Gene Therapy and Stem cells Session on May 3rd, 2020 from 3:00PM to 4:45PM EDT by Christopher D. Riemann, M.D., Vitreoretinal Surgeon and Fellowship Director, Cincinnati Eye Institute and University of Cincinnati School of Medicine; Clinical Governance Board, Cincinnati Eye Institute (presentation number 865). The presentation will provide updated data from patient cohorts 1 through 4 of the clinical study and will include data on the first patients dosed with both a new subretinal delivery system as well as with a new Thaw-and-Inject (TAI) formulation of OpRegen.

"We continue to be encouraged by positive data with OpRegen for the treatment of dry AMD," stated Brian M. Culley, CEO of Lineage. "The five patients treated as part of cohort 4, which more closely match our intended patient population, have all demonstrated an increase in the number of letters they can read on an Early Treatment Diabetic Retinopathy Scale (ETDRS), having gained between 10 25 letters. Importantly, the first patient treated using both a new subretinal delivery system and our TAI formulation of OpRegen demonstrated notable improvements in vision, having gained 25 readable letters (or 5 lines) 6 months following administration of OpRegen RPE cells, as assessed by the ETDRS. This represents an improvement in visual acuity from a baseline of 20/250 to 20/100 in the treated eye. These visual acuity measurements are meaningful and can translate into quality of life enhancements to things like reading, driving, or avoiding accidents. With the opening of two leading ophthalmology research centers as clinical sites for our study, we are focused on rapid enrollment so that our clinical update at ARVO can be as mature and informative as possible. Our objective is to combine the best cells, the best production process and the best delivery system, which we believe will position us as the front-runner in the race to address the unmet opportunity in the potential billion-dollar dry AMD market."

In addition, Lineage will present new preclinical results from its Vision Restoration Program, a proprietary program based on the ability to generate 3-dimensional human retinal tissue derived from pluripotent cells. Lineages 3-dimensional retinal tissue technology may address the unmet need of implementing a retinal tissue restoration strategy to address a wide range of severe retinal degenerative conditions including retinitis pigmentosa and advanced forms of AMD. In 2017 and 2019, the Small Business Innovation Research program of the National Institutes of Health awarded Lineage grants of close to $2.3 million to further develop this innovative, next generation vision restoration program.

- The poster presentation, entitled, "Transplantation of organoid-derived human retinal tissue in to the subretinal space of CrxRdy/+ cats)," will be presented as part of the Animal models for visual disease and restoration Session on May 4th, 2020 4:00PM to 5:45PM EDT in Session Number 291 by Igor Nasonkin, Ph.D., Principal Investigator, Director of Research & Development at Lineage (Poster board Number: 2253 - B0162).

- The poster presentation, entitled, " Intraocular biocompatibility of Hystem hydrogel for delivery of pharmaceutical agents and cells," will be presented as part of the Stem cells and organoids: Technical advances Session on May 5th, 2020 between 8:45AM to 10:30AM EDT in Session Number 332 by our collaborator Randolph D. Glickman, Ph.D., Professor of Ophthalmology, UT Health San Antonio (Poster board Number: # A0247).

Story continues

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally-differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed either to replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical programs are in markets with billion dollar opportunities and include (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase I/IIa development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase I/IIa development for the treatment of acute spinal cord injuries; and (iii) VAC2, an allogeneic cancer immunotherapy of antigen-presenting dendritic cells currently in Phase I development for the treatment of non-small cell lung cancer. Lineage is also evaluating potential partnership opportunities for Renevia, a facial aesthetics product that was recently granted a Conformit Europenne (CE) Mark. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

Forward-Looking Statements

Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as "believe," "may," "will," "estimate," "continue," "anticipate," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would," "contemplate," project," "target," "tend to," or the negative version of these words and similar expressions. Such statements include, but are not limited to, statements relating to the potential applications in Lineages Vision Restoration Program. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineages actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including risks and uncertainties inherent in Lineages business and other risks in Lineages filings with the Securities and Exchange Commission (the SEC). Lineages forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading "Risk Factors" in Lineages periodic reports with the SEC, including Lineages Annual Report on Form 10-K filed with the SEC on March 14, 2019 and its other reports, which are available from the SECs website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200218005395/en/

Contacts

Lineage Cell Therapeutics, Inc. IR Ioana C. Hone(ir@lineagecell.com) (510) 871-4188

Solebury Trout IR Gitanjali Jain Ogawa(Gogawa@troutgroup.com)(646) 378-2949

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Lineage Cell Therapeutics to Present New Data From OpRegen and Vision Restoration Programs at the Association for Research in Vision and Ophthalmology...

Spinal Cord Stem Cells Comments Off on Lineage Cell Therapeutics to Present New Data From OpRegen and Vision Restoration Programs at the Association for Research in Vision and Ophthalmology… | February 20th, 2020

Medical treatment involving stem cells is an ever-growing, billion-dollar industry, so chances are you have heard about it in the news. Here in the U.S. and around the world, stem cells are being used in various therapies to treat a wide variety of health problems and diseases, including dementia, autism, multiple sclerosis, cerebral palsy, osteoarthritis, cancer, heart disease, Parkinsons disease, and spinal cord injury. Treatments for such health issues may sound promising, but the risk is many of those being sold and advertised arent yet proven to be safe and effective. This is why its so important to educate yourself before jumping into any kind of stem cell treatment.

What are stem cells?

To gain a better understanding of this new age of medical research, one must first understand what stem cells are and how they work. Stem cells are special human cells that can develop into many different types of cells. They can divide and produce more of the same type of stem cells, or they can turn into different functioning cells. There are no other types of cells in the body that have this natural ability to generate new cell types.

Where do stem cells come from?

So where do stem cells that are used for research and medical treatments come from? The three main types of stem cells are embryonic (or pluripotent) stem cells, adult stem cells, and induced pluripotent stem cells.

Embryonic stem cells come from unused, in vitro fertilized embryos that are three to five days old. The embryos are only donated for research purposes with the informed consent of the donors. Embryonic stem cells are pluripotent, which means they can turn into any cell type in the body.

Adult stem cells are found in small numbers in developed tissues in different parts of the body, such as bone marrow, skin, and the brain. They are specific to a certain kind of tissue in the body and are limited to maintaining and repairing the tissue in which they are found. For example, liver stem cells can only make new liver tissue; they arent able to make new muscle tissue.

Induced pluripotent stem cells are another form of adult stem cells. These are stem cells that have been manipulated in a laboratory and reprogrammed to work like embryotic (or pluripotent) stem cells. While these altered adult stem cells dont appear to be clinically different from embryonic stem cells, research is still being conducted to determine if the effects they have on humans differ from actual embryonic stem cells.

Stem cells can also be found in amniotic fluid and umbilical cord blood. These stem cells have the ability to change into specialized cells like embryonic stem cells. While more research is being conducted to determine the potential of these types of stem cells, researchers already actively use these through amniocentesis procedures. In this procedure, the stem cells drawn from amniotic fluid samples of pregnant women can be screened for developmental abnormalities in a fetus.

How stem cells function

The main difference between embryonic and adult stem cells is how they function. Embryonic stem cells are more versatile. Since they can divide into more stem cells or become any type of cell in the body, they can be used to regenerate or repair a variety of diseased tissue and organs. Adult stem cells only generate the types of cells from where they are taken from in the body.

The future of stem cell research

The ability for stem cells to regenerate under the right conditions in the body or in a laboratory is why researchers and doctors have become so interested in studying them. Stem cell research is helping scientists and doctors to better understand how certain diseases occur, how to possibly generate healthy cells to replace diseased cells, and offer ways to test new drugs.

Clearly, stem cell research is showing great potential for understanding and treating a range of diseases and other health issues, but there is still a lot to learn. While there are some diseases that are showing success using stem cell treatments, many others are yet to be proven in clinical trials and should be considered highly experimental.

In our next article, various stem cell treatments, FDA regulations, and other stem cell hot topics will be explored. It will also focus on what to look for when considering stem cell therapies so people arent misled or misinformed about the benefits and risks.

For more information regarding the basics of stem cells visit these sites:

https://stemcells.nih.gov/info/basics/1.htm

https://www.mayoclinic.org/tests-procedures/bone-marrow-transplant/in-depth/stem-cells/art-20048117

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The 411 on Stem Cells: What They Are and Why It's Important to Be Educated - Legal Examiner

Spinal Cord Stem Cells Comments Off on The 411 on Stem Cells: What They Are and Why It’s Important to Be Educated – Legal Examiner | February 17th, 2020

TMRR, in its recent market report, suggests that the Induced Pluripotent Stem Cells market report is set to exceed US$ xx Mn/Bn by 2029. The report finds that the Induced Pluripotent Stem Cells market registered ~US$ xx Mn/Bn in 2018 and is spectated to grow at a healthy CAGR over the foreseeable period. This Induced Pluripotent Stem Cells market study considers 2018 as the base year, 2019 as the estimated year, and 2019 2029 as the forecast timeframe.

The Induced Pluripotent Stem Cells market research focuses on the market structure and various factors (positive and negative) affecting the growth of the market. The study encloses a precise evaluation of the Induced Pluripotent Stem Cells market, including growth rate, current scenario, and volume inflation prospects, on the basis of DROT and Porters Five Forces analyses. In addition, the Induced Pluripotent Stem Cells market study provides reliable and authentic projections regarding the technical jargon.

Important regions covered in the Induced Pluripotent Stem Cells market research include Region 1 (Country 1, country 2), Region 2 (Country 1, country 2), Region 3 (Country 1, country 2) and Region 4 (Country 1, country 2).

Request For Discount On This Report @ https://www.tmrresearch.com/sample/sample?flag=D&rep_id=6245&source=atm

The Induced Pluripotent Stem Cells market study answers critical questions including:

The content of the Induced Pluripotent Stem Cells market report includes the following insights:

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On the basis of component, the global Induced Pluripotent Stem Cells market report covers the following segments:

Notable Development

Over the past few years, fast emerging markets in the global induced pluripotent stem cells are seeing the advent of patents that unveil new techniques for reprogramming of adult cells to reach embryonic stage. Particularly, the idea that these pluripotent stem cells can be made to form any cells in the body has galvanized companies to test their potential in human cell lines. Also, a few biotech companies have intensified their research efforts to improve the safety of and reduce the risk of genetic aberrations in their approved human cell lines. Recently, this has seen the form of collaborative efforts among them.

Lineage Cell Therapeutics and AgeX Therapeutics have in December 2019 announced that they have applied for a patent for a new method for generating iPSCs. These are based on NIH-approved human cell lines, and have been undergoing clinical-stage programs in the treatment of dry macular degeneration and spinal cord injuries. The companies claim to include multiple techniques for reprogramming of animal somatic cells.

Such initiatives by biotech companies are expected to impart a solid push to the evolution of the induced pluripotent stem cells.

North America is one of the regions attracting colossal research funding and industry investments in induced pluripotent stem cells technologies. Continuous efforts of players to generate immune-matched supply of pluripotent cells to be used in disease modelling has been a key accelerator for growth. Meanwhile, Asia Pacific has also been showing a promising potential in the expansion of the prospects of the market. The rising number of programs for expanding stem cell-based therapy is opening new avenues in the market.

All the players running in the global Induced Pluripotent Stem Cells market are elaborated thoroughly in the Induced Pluripotent Stem Cells market report on the basis of R&D developments, distribution channels, industrial penetration, manufacturing processes, and revenue. In addition, the report examines, legal policies, and comparative analysis between the leading and emerging Induced Pluripotent Stem Cells market players.

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Tags: Induced Pluripotent SteInduced Pluripotent Stem Cells Market Definitions and OverviewInduced Pluripotent Stem Cells Market DynamicsInduced Pluripotent Stem Cells Market Segmentation and Scope

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Induced Pluripotent Stem Cells Market Predicted to Witness Surge in the Near Future2018 2028 - TechNews.mobi

Spinal Cord Stem Cells Comments Off on Induced Pluripotent Stem Cells Market Predicted to Witness Surge in the Near Future2018 2028 – TechNews.mobi | February 14th, 2020

Motor neuron disease is a degenerative condition which destroys the nerve cells (motor neurons) in the brain and spinal cord, which control movement, speech, swallowing and breathing. The most common type of motor neuron disease is amyotrophic lateral sclerosis (ALS), which affects around 5,000 people in the UK at any one time.A new study found that in this disease, the motor neurons in the brain and spinal cord become sick and die when a protein, called TDP-43, misfolds and accumulates in the wrong place within the motor neurons. Conversely, when this happens in a type of cell that supports motor neurons, called astrocytes, these cells appear comparatively resistant and survive.

When these two types of cells are close together, the more-resistant astrocytes are able to protect motor neurons from the misfolded protein. This rescue-mechanism helps the motor neurons, which are needed to control muscles, live longer.

The role astrocytes have played in dealing with toxic forms of TDP-43 in motor neurons has not been previously well documented in motor neuron disease. Its exciting that weve now found that they may play an important protective role in the early-stages of this disease, explains Phillip Smethurst, lead author. This has huge therapeutic potential finding ways to harness the protective properties of astrocytes could pave the way to new treatments. This could prolong their rescue function or find a way to mimic their behavior in motor neurons so that they can protect themselves from the toxic protein.

This research also established a new model for studying motor neuron disease. This new method more closely resembles the disease in patients as it uses healthy human stem cells, derived from skin cells, and spinal cord tissue samples donated by patients with motor neuron disease, collected post-mortem.

It is thanks to the selfless donations from people with motor neuron disease, that we were able to study the interplay between motor neurons and astrocytes in conditions that closely resemble what happens in humans. These human cell models are a powerful tool for further studies of motor neuron disease and in the hunt for effective therapies. explains Katie Sidle, co-senior author.

For the first time, we have been able to create a model of sporadic motor neuron disease by essentially transferring the toxic TDP-43 protein from post-mortem tissue into healthy human stem cell-derived motor neurons and astrocytes in order to understand how each cell type responds to this insult, both in isolation and when mixed together. The insights made in this work are testament to the power of creative collaboration and interdisciplinarity. It is through many years working together as a group of clinicians, pathologists, stem cell biologists, protein biochemists and other experts, and with a joint aim of increasing knowledge about motor neuron disease (to ultimately help find a cure), that these results have been possible, says Rickie Patani, co-senior author.ReferenceSmethurst et al. (2020) Distinct responses of neurons and astrocytes to TDP-43 proteinopathy in amyotrophic lateral sclerosis. Brain. DOI: https://doi.org/10.1093/brain/awz419

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

Link:
Astrocytes Show Protective Role in Early-stage ALS - Technology Networks

Spinal Cord Stem Cells Comments Off on Astrocytes Show Protective Role in Early-stage ALS – Technology Networks | February 13th, 2020

Scientists using a new motor neuron disease (MND) model have shown astrocytes may protect neurons from toxic TDP-43 protein aggregates in the early stages of disease.

Researchers have discovered that astrocytes can protect motor neurons in the central nervous system (CNS) from the toxicity of misfolded protein, TDP-43, in sporadic motor neuron disease (MND). The team suggest this rescue mechanism could be harnessed to slow disease progression, particularly in amyotrophic lateral sclerosis (ALS).

The study, published in Brain, demonstrated that this neurodegenerative disease is caused by accumulation of TDP-43 in motor neurons, resulting in cell death. However, the scientists noted that TDP-43 accumulation in neural support cells, called astrocytes, does not cause death. Instead they appear comparatively resistant.

According to the paper, when the two cell types are together, astrocytes protect motor neurons from the protein aggregates, promoting their survival. The researchers from the Francis Crick Institute and University College London, both UK, suggest that these cells may therefore be supporting motor neurons early on in sporadic MND. They called this a rescue mechanism.

when the two cell types are together, astrocytes protect motor neurons from the protein aggregates, promoting their survival

The role astrocytes have played in dealing with toxic forms of TDP-43 in motor neurons has not been previously well documented in motor neuron disease. Its exciting that weve now found that they may play an important protective role in the early-stages of this disease, explains Phillip Smethurst, lead author and former postdoc in the Human Stem Cells and Neurodegeneration Laboratory at the Crick. This has huge therapeutic potential finding ways to harness the protective properties of astrocytes could pave the way to new treatments. This could prolong their rescue function or find a way to mimic their behaviour in motor neurons so that they can protect themselves from the toxic protein.

In order to conduct this research, the team created a new model for MND, which more closely resembles the disease in patients. In the model they took healthy adult stem cells and exposed them to the toxic TPD-43 protein using post-mortem spinal cord tissue samples donated by patients with MND.

For the first time, we have been able to create a model of sporadic motor neuron disease by essentially transferring the toxic TDP-43 protein from post-mortem tissue into healthy human stem cell-derived motor neurons and astrocytes in order to understand how each cell type responds to this insult, both in isolation and when mixed together, said Dr Rickie Patani, co-senior author, group leader of the Human Stem Cells and Neurodegeneration Laboratory at the Crick and Professor of Human Stem Cells and Regenerative Neurology at UCL Queen Square Institute of Neurology.

Read the original:
Astrocytes could be harnessed to protect motor neurons in MND - Drug Target Review

Spinal Cord Stem Cells Comments Off on Astrocytes could be harnessed to protect motor neurons in MND – Drug Target Review | February 11th, 2020

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Newswise A UCLA-led study reveals a new role for a gene thats associated with autism spectrum disorder, intellectual disability and language impairment.

The gene, Foxp1, has previously been studied for its function in the neurons of the developing brain. But the new study reveals that its also important in a group of brain stem cells the precursors to mature neurons.

This discovery really broadens the scope of where we think Foxp1 is important, said Bennett Novitch, a member of theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLAand the senior author of the paper. And this gives us an expanded way of thinking about how its mutation affects patients.

Mutations in Foxp1 were first identified in patients with autism and language impairments more than a decade ago. During embryonic development, the protein plays a broad role in controlling the activity of many other genes related to blood, lung, heart, brain and spinal cord development. To study how Foxp1 mutations might cause autism, researchers have typically analyzed its role in the brains neurons.

Almost all of the attention has been placed on the expression of Foxp1 in neurons that are already formed, said Novitch, a UCLA professor of neurobiology who holds the Ethel Scheibel Chair in Neuroscience.

In the new study published in Cell Reports, he and his colleagues monitored levels of Foxp1 in the brains of developing mouse embryos. They found that, in normally developing animals, the gene was active far earlier than previous studies have indicated during the period when neural stem cells known as apical radial glia were just beginning to expand in numbers and generate a subset of brain cells found deep within the developing brain.

When mice lacked Foxp1, however, there were fewer apical radial glia at early stages of brain development, as well as fewer of the deep brain cells they normally produce. When levels of Foxp1 were above normal, the researchers observed more apical radial glia and an excess of those deep brain cells that appear early in development.In addition, continued high levels of Foxp1 at later stages of embryonic development led to unusual patterns of apical radial glia production of deep-layer neurons even after the mice were born.

What we saw was that both too much and too little Foxp1 affects the ability of neural stem cells to replicate and form certain neurons in a specific sequence in mice, Novitch said. And this fits with the structural and behavioral abnormalities that have been seen in human patients.

Some people, he explained, have mutations in the Foxp1 gene that blunt the activity of the Foxp1 protein, while others have mutations that change the proteins structure or make it hyperactive.

The team also found intriguing hints that Foxp1 might be important for a property specific to the developing human brain.The researchers also examined human brain tissue and discovered that Foxp1 is present not only in apical radial glia, as was seen in mice, but also in a second group of neuralstem cells called basal radial glia.

Basal radial glia are abundant in the developinghuman brain, but absent or sparse in the brains of many other animals, including mice.However, when Novitchs team elevated Foxp1 function in the brains of mice, cells resembling basal radial glia were formed. Scientists have hypothesized that basal radial glia also are connected to the size of the human brain cortex: Their presence in large quantities in the human brain may help explain why it is disproportionately larger than those of other animals.

Novitch said that although the new research does not have any immediate implications for the treatment of autism or other diseases associated with Foxp1 mutations, it does help researchers understand the underlying causes of those disorders.

In future research, Novitch and his colleagues are planning to study what genes Foxp1 regulates in apical radial glia and basal radial glia, and what roles those genes play in the developing brain.

The studys first author is Caroline Alayne Pearson, a UCLA assistant project scientist. Other authors are from the University of Texas at Austin, the University of Alabama at Birmingham and the University of Puerto Rico.

The study was funded by the National Institutes of Health, the California Institute for Regenerative Medicine, the Cancer Prevention and Research Institute of Texas, the University of Texas at Austins Marie Betzner Morrow Centennial Endowment and the UCLA Broad Stem Cell Research Centers Research Award Program, including support from the Binder Foundation.

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Gene associated with autism also controls growth of the embryonic brain - Newswise

Spinal Cord Stem Cells Comments Off on Gene associated with autism also controls growth of the embryonic brain – Newswise | February 11th, 2020

Methionine is an amino acid found in meat, eggs, and dairy. Its absorbed by T-cells that are part of our immune system. Those cells are also believed to be the immune cells that attack our myelin, creating the nerve damage that results in multiple sclerosis.

In this study, mice eating less methionine had a reduced number of a certain type of T-cell, which led to a delay in disease onset and progression. The researchers believe reducing methionine intake can actually dampen the immune cells that cause disease, leading to better outcomes.

Changing a persons diet to reduce the amount of methionine (amino acid found in food) could delay the development and progression of inflammatory and autoimmune disorders, including multiple sclerosis (MS).

That finding was described in the study Methionine Metabolism Shapes T Helper Cell Responses through Regulation of Epigenetic Reprogramming, published recently in the journal Cell Metabolism.

Click here to read the full story.

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Unlike hematopoietic stem cell transplants, in which stem cells are removed from a patients bone marrow and later infused back into the bloodstream, mesenchymal stem cell transplants (MSCT) collect those stem cells from the patients spinal column and return them there. This study concludes that MSCT is safe and that cells delivered into the spinal cord produced a significantly slower disease progression rate than did cells delivered into the bloodstream.

Transplanting patients ownmesenchymal stem cellsis a safe therapeutic approach and can delay disease progression in people with MS, a meta-analysis review shows.

The study also showed that cells transplanted to the spinal cord (intrathecal injection) were associated with significantly slower disease progression rates, compared to cells delivered into the bloodstream.

Click here to read the full story.

***

Why do neurologists often use spinal taps when determining whether someone has MS? This study provides one of the reasons.

People with MS have a more diverse set of immune cells in their cerebrospinal fluid (CSF), the fluid that bathes the central nervous system, but no such diversity is seen in their blood, a study reports. Instead, MS causes changes in the activation of immune cells in the blood.

The distinct set of immune cells in MS patients CSF shows enrichment of pro-inflammatory cells that promote disease severity in MS mouse models.

Click here to read the full story.

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Heres encouraging news about a possible treatment that can lower the number of brain lesions in someone with MS. Keep in mind this is only a Phase 2 trial. A Phase 3 trial isnt expected until later this year. However, a news release from research sponsor Sanofisays, This molecule may be the first B-cell-targeted MS therapy that not only inhibits the peripheral immune system, but also crosses the blood-brain barrier to suppress immune cells that have migrated into the brain.

The experimental BTK inhibitor SAR442168 showed an acceptable safety profile and met its primary endpoint a significant reduction in the number of new lesions visible on a brain imaging scan in a Phase 2 trial in people with MS, study results show.

SAR442168, formerly known as PRN2246, is an oral, small molecule being co-developed by Principia Biopharmaand Sanofi Genzyme. It works by inhibiting Brutons tyrosine kinase (BTK), a protein important for the proliferation of immune cells, particularly B-cells. By blocking BTK, it is expected that SAR442168 can reduce inflammation that damages the nervous system in people with MS.

Click here to read the full story.

Did you know that some of my columns from The MS Wire are now available as audio briefings? You can listen to them here.

***

Note: Multiple Sclerosis News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Multiple Sclerosis News Today or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to multiple sclerosis.

Ed Tobias is a retired broadcast journalist. Most of his 40+ year career was spent as a manager with the Associated Press in Washington, DC. Tobias was diagnosed with Multiple Sclerosis in 1980 but he continued to work, full-time, meeting interesting people and traveling to interesting places, until retiring at the end of 2012.

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MS News that Caught My Eye Last Week: Methionine, MSCT, Spinal... - Multiple Sclerosis News Today

Spinal Cord Stem Cells Comments Off on MS News that Caught My Eye Last Week: Methionine, MSCT, Spinal… – Multiple Sclerosis News Today | February 10th, 2020

SpringWorks Therapeutics (NASDAQ:SWTX) and Neuralstem (NASDAQ:CUR) are both small-cap medical companies, but which is the better business? We will contrast the two companies based on the strength of their profitability, dividends, institutional ownership, valuation, earnings, analyst recommendations and risk.

Analyst Recommendations

This is a summary of recent ratings and recommmendations for SpringWorks Therapeutics and Neuralstem, as reported by MarketBeat.

SpringWorks Therapeutics presently has a consensus target price of $35.50, indicating a potential upside of 7.51%. Given SpringWorks Therapeutics higher possible upside, analysts clearly believe SpringWorks Therapeutics is more favorable than Neuralstem.

Profitability

This table compares SpringWorks Therapeutics and Neuralstems net margins, return on equity and return on assets.

Insider and Institutional Ownership

72.2% of SpringWorks Therapeutics shares are owned by institutional investors. Comparatively, 38.3% of Neuralstem shares are owned by institutional investors. 5.4% of Neuralstem shares are owned by company insiders. Strong institutional ownership is an indication that endowments, large money managers and hedge funds believe a company is poised for long-term growth.

Valuation and Earnings

This table compares SpringWorks Therapeutics and Neuralstems revenue, earnings per share (EPS) and valuation.

SpringWorks Therapeutics has higher earnings, but lower revenue than Neuralstem.

Summary

SpringWorks Therapeutics beats Neuralstem on 6 of the 8 factors compared between the two stocks.

SpringWorks Therapeutics Company Profile

SpringWorks Therapeutics, Inc., a clinical-stage biopharmaceutical company, acquires, develops, and commercializes medicines for underserved patient populations suffering from rare diseases and cancer. Its advanced product candidate is nirogacestat, an oral small molecule gamma secretase inhibitor that is in Phase 3 clinical trials for the treatment of desmoid tumors. The company is also developing mirdametinib, an oral small molecule MEK inhibitor that is in Phase 2b clinical trials for the treatment of neurofibromatosis type 1-associated plexiform neurofibromas; and Nirogacestat + belantamab mafodotin, which is in Phase 1b clinical trials for the treatment of relapsed or refractory multiple myeloma. In addition, it is developing Mirdametinib + lifirafenib, a combination therapy that is in Phase 1b clinical trials in patients with advanced or refractory solid tumors; and BGB-3245, an investigational oral selective small molecule inhibitor of specific BRAF driver mutations and genetic fusions, which is in preclinical studies in a range of tumor models with BRAF mutations or fusions. The company has collaborations with BeiGene, Ltd. and GlaxoSmithKline plc to develop combination approaches with nirogacestat and mirdametinib, as well as other standalone medicines. SpringWorks Therapeutics, Inc. was founded in 2017 and is headquartered in Stamford, Connecticut.

Neuralstem Company Profile

Neuralstem, Inc., a clinical stage biopharmaceutical company, focuses on the research and development of nervous system therapies based on its proprietary human neuronal stem cells and small molecule compounds. The company's stem cell based technology enables the isolation and expansion of human neural stem cells from various areas of the developing human brain and spinal cord enabling the generation of physiologically relevant human neurons of various types. Its lead product candidate is NSI-189, a chemical entity, which has been completed Phase II clinical trial for the treatment of major depressive disorder, as well as is in preclinical study for the treatment-refractory depression, Angelman Syndrome, Alzheimer's disease, ischemic stroke, diabetic neuropathy, irradiation-induced cognitive deficit, and long-term potentiation enhancement. The company also develops NSI-566, which has completed Phase II clinical trial for treating amyotrophic lateral sclerosis disease; Phase II clinical trial for the treatment of chronic ischemic stroke; and Phase I clinical trials for the treatment of chronic spinal cord injury, as well as is in preclinical study for the traumatic brain injury. In addition, it develops NSI-532, which is in preclinical study for treatment of Alzheimer's disease; and NSI-777 that is in preclinical study for treatment of human demyelinating diseases. Neuralstem, Inc. was founded in 1996 and is headquartered in Germantown, Maryland.

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Contrasting Neuralstem (NASDAQ:CUR) and SpringWorks Therapeutics (NASDAQ:SWTX) - Riverton Roll

Spinal Cord Stem Cells Comments Off on Contrasting Neuralstem (NASDAQ:CUR) and SpringWorks Therapeutics (NASDAQ:SWTX) – Riverton Roll | February 9th, 2020

Autolus Therapeutics (NASDAQ:AUTL) and Neuralstem (NASDAQ:CUR) are both small-cap medical companies, but which is the better business? We will compare the two companies based on the strength of their profitability, analyst recommendations, dividends, earnings, risk, valuation and institutional ownership.

Profitability

This table compares Autolus Therapeutics and Neuralstems net margins, return on equity and return on assets.

Insider & Institutional Ownership

26.6% of Autolus Therapeutics shares are owned by institutional investors. Comparatively, 38.3% of Neuralstem shares are owned by institutional investors. 5.4% of Neuralstem shares are owned by insiders. Strong institutional ownership is an indication that large money managers, endowments and hedge funds believe a company will outperform the market over the long term.

Earnings & Valuation

This table compares Autolus Therapeutics and Neuralstems gross revenue, earnings per share and valuation.

Neuralstem has lower revenue, but higher earnings than Autolus Therapeutics.

Risk and Volatility

Autolus Therapeutics has a beta of 0.88, suggesting that its stock price is 12% less volatile than the S&P 500. Comparatively, Neuralstem has a beta of 1.81, suggesting that its stock price is 81% more volatile than the S&P 500.

Analyst Ratings

This is a summary of current ratings and recommmendations for Autolus Therapeutics and Neuralstem, as provided by MarketBeat.

Autolus Therapeutics currently has a consensus target price of $25.00, suggesting a potential upside of 155.10%. Given Autolus Therapeutics higher probable upside, equities analysts plainly believe Autolus Therapeutics is more favorable than Neuralstem.

Summary

Autolus Therapeutics beats Neuralstem on 7 of the 11 factors compared between the two stocks.

About Autolus Therapeutics

Autolus Therapeutics plc, a biopharmaceutical company, develops T cell therapies for the treatment of cancer. The company is developing AUTO1, a CD19-targeting programmed T cell therapy, which is in Phase I trial to reduce the risk of severe cytokine release syndrome; AUTO2, a dual-targeting programmed T cell therapy that is in Phase I/II clinical trial for the treatment of relapsed or refractory multiple myeloma; and AUTO3, a dual-targeting programmed T cell therapy, which is in Phase I/II clinical trials for treating relapsed or refractory diffuse large B-cell lymphoma. It is also developing AUTO4, a programmed T cell therapy that is in Phase I/II clinical trial for the treatment of peripheral T-cell lymphoma; and AUTO6, a programmed T cell therapy for treating neuroblastoma. Autolus Therapeutics plc has a collaboration partnership with AbCellera Biologics Inc. on antibody discovery project. The company was founded in 2014 and is headquartered in London, the United Kingdom.

About Neuralstem

Neuralstem, Inc., a clinical stage biopharmaceutical company, focuses on the research and development of nervous system therapies based on its proprietary human neuronal stem cells and small molecule compounds. The company's stem cell based technology enables the isolation and expansion of human neural stem cells from various areas of the developing human brain and spinal cord enabling the generation of physiologically relevant human neurons of various types. Its lead product candidate is NSI-189, a chemical entity, which has been completed Phase II clinical trial for the treatment of major depressive disorder, as well as is in preclinical study for the treatment-refractory depression, Angelman Syndrome, Alzheimer's disease, ischemic stroke, diabetic neuropathy, irradiation-induced cognitive deficit, and long-term potentiation enhancement. The company also develops NSI-566, which has completed Phase II clinical trial for treating amyotrophic lateral sclerosis disease; Phase II clinical trial for the treatment of chronic ischemic stroke; and Phase I clinical trials for the treatment of chronic spinal cord injury, as well as is in preclinical study for the traumatic brain injury. In addition, it develops NSI-532, which is in preclinical study for treatment of Alzheimer's disease; and NSI-777 that is in preclinical study for treatment of human demyelinating diseases. Neuralstem, Inc. was founded in 1996 and is headquartered in Germantown, Maryland.

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Head to Head Review: Autolus Therapeutics (NASDAQ:AUTL) and Neuralstem (NASDAQ:CUR) - Riverton Roll

Spinal Cord Stem Cells Comments Off on Head to Head Review: Autolus Therapeutics (NASDAQ:AUTL) and Neuralstem (NASDAQ:CUR) – Riverton Roll | February 8th, 2020