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

By daniellenierenberg

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

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

By daniellenierenberg

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

By daniellenierenberg

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

By daniellenierenberg

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

By daniellenierenberg

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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Why choose TMRR?

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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Astrocytes Show Protective Role in Early-stage ALS – Technology Networks

By daniellenierenberg

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.

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

By daniellenierenberg

MEDIA CONTACT

Available for logged-in reporters only

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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Astrocytes could be harnessed to protect motor neurons in MND – Drug Target Review

By daniellenierenberg

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.

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

By daniellenierenberg

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.

***

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.

***

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

By daniellenierenberg

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

By daniellenierenberg

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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Abnormal Bone Formation After Trauma Explained and Reversed in Mice – Michigan Medicine

By daniellenierenberg

Hip replacements, severe burns, spinal cord injuries, blast injuries, traumatic brain injuriesthese seemingly disparate traumas can each lead to a painful complication during the healing process called heterotopic ossification. Heterotopic ossification is abnormal bone formation within muscle and soft tissues, an unfortunately common phenomenon that typically occurs weeks after an injury or surgery. Patients with heterotopic ossification experience decreased range of motion, swelling and pain.

Currently, theres no way to prevent it and once its formed, theres no way to reverse it, says Benjamin Levi, M.D., Director of the Burn/Wound/Regeneration Medicine Laboratory and Center for Basic and Translational Research in Michigan Medicines Department of Surgery. And while experts suspected that heterotopic ossification was somehow linked to inflammation, new U-M research explains how this happens on a cellular scaleand suggests a way it can be stopped.

To help explain how the healing process goes awry in heterotopic ossification, the research team, led by Levi, Michael Sorkin, M.D. and Amanda Huber, Ph.D., of the Department of Surgerys section of plastic surgery, took a closer look at the inflammation process in mice. Using tissue from injury sites in mouse models of heterotopic ossification, they used single cell RNA sequencing to characterize the types of cells present. They confirmed that macrophages were among the first responders and might be behind aberrant healing.

Macrophages are white blood cells whose normal job is to find and destroy pathogens. Upon closer examination, the Michigan team found that macrophages are more complex than previously thoughtand dont always do what they are supposed to do.

Macrophages are a heterogenous population, some that are helpful with healing and some that are not, explains Levi. People think of macrophages as binary (M1 vs. M2). Yet weve shown that there are many different macrophage phenotypes or states that are present during abnormal wound healing.

Specifically, during heterotopic ossification formation, the increased presence of macrophages that express TGF-beta leads to an errant signal being sent to bone forming stem cells.

For now, the only way to treat heterotopic ossification is to wait for it to stop growing and cut it out which never completely restores joint function. This new research suggests that there may be a way to treat it at the cellular level. Working with the lab led by Stephen Kunkel, Ph.D. of the Department of Pathology, the team demonstrated that an activating peptide to CD47, p7N3 could alter TGF-beta expressing macrophages, reducing their ability to send signals to bone-forming stem cells that lead to heterotopic ossification.

During abnormal wound healing, we think there is some signal that continues to be present at an injury site even after the injury should have resolved, says Levi. Beyond heterotopic ossification, Levi says the studys findings can likely be translated to other types of abnormal wound healing like muscle fibrosis.

The team hopes to eventually develop translational therapies that target this pathway and further characterize not just the inflammatory cells but the stem cells responsible for the abnormal bone formation.

The paper is published in the journal Nature Communications. Other U-M authors include: Charles Hwang, William Carson IV, Rajarsee Menon, John Li, Kaetlin Vasquez, Chase Pagani, Nicole Patel, Shuli Li, Noelle D. Visser, Yashar Niknafs, Shawn Loder, Melissa Scola, Dylan Nycz, Katherine Gallagher, Laurie K. McCauley, Shailesh Agarwal, and Yuji Mishina.

Paper Cited: Regulation of heterotopic ossification by monocytes in a mouse model of aberrant wound healing, Nature Communications, DOI: 10.1038/s41467-019-14172-4

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Yes, I’ve seen the stories about that new spinal treatment. Here’s why I’m not interested – CBC.ca

By daniellenierenberg

It happens whenever there are news stories about a new treatment claiming to cure paralysis.

I get flooded with messages from distant relatives, or people I went to high school with but haven't seen in 10 years.

"OMG YOU TOTALLY NEED TO DO THIS."

I'm not saying other people shouldn't try whatever treatments they want to, but for me, there's too little certainty and too many unknown factors.

After 15 years in a wheelchair, I've gained the perspective that walking, in fact, does not equate happiness.

I'll never forget the metaphor used to explain to me how one would repair a spinal cord, even if I was a little high on morphine when I heard it.

I was 16, laying in a hospital bed with four large screws drilled into my skull to stabilize my spine.

"Imagine squeezing all of the toothpaste out of a tube. Now try and get that toothpaste back into the tube without changing it's shape or structure. That's how fragile your spinal cord is."

Sounds impossible, right? Maybe. Or maybe the technology just hasn't been invented yet.

The thing no one tells you about having a spinal cord injury is that not walking is the easiest adjustment. You don't need to check your eyes. You read that right.

The human body and muscle memory are pretty adaptable. Using a wheelchair is the easy part.

First, there are societal stigmas. They could fill their own novel.

Every person with a disability has a few horror stories. Personally, I applied to more than 600 jobs before getting a part-time, entry-level position. Shout out to the YMCA of Saskatoon for giving me a shot when no one else would!

Another thing that doesn't often get talked about is the secondary health issues that come along with a spinal cord injury. Low blood pressure, autonomic dysreflexia, inability to regulate your body temperature, bowel and bladder problems, and pressure sores to name a few.

These are the really hard parts.

These secondary health issues are why I have no interest in an epidural stimulation implant or any other elective surgery of that nature. I know it's exciting to see someone moving their leg after an injury or walking while assisted, but the truth is we don't know what other unknown factors such treatments might present.

I've seen plenty of stories about possible "cures." When I was first injured it was stem cells, then embryonic stem cells, then there were the paralyzed rats learning to walk again.

Every few years there's a new procedure that makes a splash. Everyone is positive that this time, this procedure, this one is the cure. None have come to fruition.

Hope and optimism are vital, but there's a fine line between hope and false hope. I've seen far too many people unable to overcome the false hope and remain bitter and angry that they can't be "normal."

The thing is, moving my leg or even walking (although it would be cool) wouldn't change the functional quality of my life. I'm already independent. I'm already quite happy with my life. Gaining the ability to move a leg still wouldn't address those secondary health issues.

I'm not saying any of these potential treatments aren't amazing advancements in medicine, but if you can't guarantee me the ability to sweat so I don't overheat in our prairie summers, or control of my bowels or bladder, it's not worth the unknowns or cost at this point in my life.

There's no guarantee. I won't compromise for a maybe.

This column is part of CBC'sOpinionsection. For more information about this section, please read thiseditor's blogand ourFAQ.

Interested in writing for us? We accept pitches for opinion and point-of-view pieces from Saskatchewan residents who want to share their thoughts on the news of the day, issues affecting their community or who have a compelling personal story to share. No need to be a professional writer!

Read more about what we're looking for here, then emailsask-opinion-grp@cbc.cawith your idea.

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Stem Cell Regeneration for Spinal Cord injuries

By daniellenierenberg

Spinal cord injuries can result in severe neurological dysfunction, including motor, sensory, and autonomic paralysis, and up until now there has been no cure or effective treatment for such injuries.

But the first human trial based on Nobel Prize winning induced pluripotent stem cells (IPSC) technology, is due to start in Japan, giving hope to hundreds of thousands of paralysed patients, that there might be light at the end of their tunnel.

The spinal cord is responsible for relaying signals up and down the body from the brain to the nervous system. The spinal cord is a bundle of nerves contained in the spinal canal, which is cocooned in the spinal column (not to be confused with the spinal cord they are two very separate entities).

The spinal cord itself has a protective sheath wrapped around it which acts as insulation whilst allowing nerve signals from the brain to travel even faster to where they need to go.

The spinal column is divided into five distinct sections:

The site of the spinal cord injury will determine the severity of the injury and injuries are classified as either:

The higher up the spinal cord the injury occurs, the more function and feeling will be lost. It is estimated that approximately every year there are between 8 to 246 cases per million incidences of spinal cord injuries worldwide.

Stem cell therapy is amongst the most exciting ongoing research for people with spinal cord injuries, in modern medicine. Because whilst the research is still in its infancy, legitimate trials are showing promising results.

According to the Journal of the American Academy of Orthopedic Surgeons, there are different stem cells which have varying abilities to restore certain functions.

Stem cells are self-renewing cells that can differentiate into one or more specific cell types. For people with spinal cord injuries, stem cells could prevent further cell death, stimulate cell growth from the existing cells and even replace the injured cells, restoring the communication channels between the body and the brain.

Until recently, stem cell research has involved looking at:

It is research into these induced pluripotent stem cells that the team in Japan are currently laying down the groundwork for. They are planning to conduct a first-in-human study of an induced pluripotent stem cell-based intervention, for subacute spinal cord injury.

Not only that, but it is the first such therapy to look into treating this kind of injury, that has ever received government approval for sale to patients.

However there are concerns by those who work in the field, but arent working on this particular project, that the evidence to support the suggestion that the treatment works, is insufficient. They state that the approval for the research was based on a small, poorly designed clinical trial.

Like the majority of scientific breakthroughs that have gone before, there will always be naysayers we used to think the world was flat and the sun orbited Earth, that the body was composed of four humours and an imbalance in those made us sick.

Those theories were disproved, and look how far weve come since then. Now imagine if we could make a paralysed person walk again. It will happen. But for now, lets celebrate and support this team for trying.

Because whilst there have been multiple attempts to develop stem cell transplantation approaches with the aim to regenerate damaged spinal cords before, this multicentre team is planning the first that might actually work, and be ethical to boot.

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SASpine to offer Stem Cell Therapy – Yahoo Finance

By daniellenierenberg

SAN ANTONIO, Feb. 3, 2020 /PRNewswire/ -- SASpine is now offering cutting edge Stem Cell Treatments to patients. For the past several years Dr. Steven Cyr, Mayo Clinic Trained Spine Surgeon, has been researching the benefits of stem cells in the treatment of multiple medical conditions including spinal disorders, specifically, conditions which involve spinal cord injury, degenerative disc disease, herniated discs, and as a supplement to enhance the success of Spinal fusions when treating instability, deformity, and fractures of the spine.

Steven J. Cyr, M.D., is a spine surgeon who has gained a reputation for surgical excellence in Texas, throughout the nation, and abroad.

Dr. Steven Cyr has been treating patients using growth factors and stem cells contained in amniotic tissue and bone marrow aspirate to provide a potential for improved success with fusion procedures, when treating herniated discs, and for arthritic or damaged joints, with remarkable success. "The goal of any medical intervention is to yield improved outcomes with the ideal result of returning a patient to normal function, when possible," states Dr Cyr. He went on to elaborate that there are times when only a structural solution can solve problems related to spinal disorders, but even in that scenario, the use of stem cells or growth factors derived from stem cell products can possibly improve the success of surgical procedures. "I have patients previously unable to jog or run return to normal function and athletic ability after injections of growth factors and stem cell products into the knee joints, hip joints, and shoulder joints," he said. "This includes high-level athletes, professional dancers, and the average weekend warrior."

There may be promise in treating patients with spinal cord injury as well. SASpine CEO, LeAnn Cyr, states, "There are reports of patients gaining significant neurological improvement after being treated with stem cells." Dr Cyr continues, "Most patients with spinal cord injuries resulting from trauma also have mechanical pressure on the nerves that result either from bone fragments or disc material compressing the spinal cord that needs to be removed along with surgical stabilization of the spinal bones. There's significant potential that stem cells bring to the equation when treating these types of patients, and I am excited about the potential that these products offer to the host of treatments to address spinal conditions and arthritic joints."

For more information about SASpine's Stem Cell Treatment Program, visit http://www.saspine.com or call (210) 487-7463 in San Antonio or (832) 919-7990 in Houston.

Related Linkswww.facebook.com/saspinewww.instagram.com/surgical.associates.in.spine

If you've been living with back pain, you're not alone. Here at SASpine, we have experienced spine specialists who are committed to improving your quality of life. (PRNewsfoto/SASpine)

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Patient in Japan 1st to have iPS cell heart muscle transplant : The Asahi Shimbun – Asahi Shimbun

By daniellenierenberg

A patient who received the worlds first transplant of cardiac muscle cells using artificially derived stem cells known as iPS cells this month is in stable condition, an Osaka University team said Jan. 27.

After surgery, doctors closely monitored the patient, who had ischemic cardiomyopathy, a condition in which clotted arteries cause heart muscles to malfunction. But the patient has been moved toa general hospital ward, the team said.

Yoshiki Sawa, a professor of cardiovascular surgery at the university, who led the team that conducted the transplant, said the team aims to put the technique into practical use.

Sawa said the team hopestransplants of heart muscle tissues derived from induced pluripotent stem cellswill be used to save many patients who have heart conditions.

In the clinical trial, three sheets of heart muscle tissues made from iPS cells stocked at Kyoto Universitys Center for iPS Cell Research and Application were attached to affected parts of the patients heart. The iPS cells were created from tissues provided by a healthy donor.

The sheets were 4 to 5 centimeters in diameter and 0.1 millimeter thick.

The transplant's goal is to regenerate cardiac blood vessels using a substance secreted by the sheets of muscle cells. The sheets are degradable and disappear from the body several months after they secrete the substance, according to the team.

The university plans to perform similar transplants on nine other patients who have serious heart problems.

The Osaka University team had planned to conduct the clinical trial of the transplant earlier after the government approved the plan in May 2018.

But it was postponed due to damage from a powerful earthquake that hit Osaka Prefecture the following month that rendered its facility to cultivate cells unusable.

The trial is part of the process toward the future distribution of medical products using cells.

Osaka Universitys announcement of the successful transplant of tissues created from iPS cells marked the fourth such transplantation.

Including Osaka University's trial, Japanese surgeons have now successfully transplanted tissues created from iPS cells four times.

The world's first transplant of iPS-derived cells was conducted in 2014 whenthe Riken research institute transplanted retina cells for a patient with age-related macular degeneration.

In 2018, Kyoto University transplanted nerve cells for a Parkinson disease patient. Osaka University transplanted cornea cells into a patient with a disease of the cornea in 2019.

Patients who undergo transplants using iPS-derived cell must accept the risk that the cells may become cancerous.

The moreiPS-derived cells a patient receives, the higher their risk.

Hundreds of thousands of retina cells were used in the 2014 retina transplant. In the 2018 and 2019 transplants, the number of nerve and cornea cells used soared to between 5 million to 6 million.

Osaka University's latest transplant utilized roughly 100 milliontissues made from iPS cells.

Sawa acknowledged the transplanted heart muscle tissues could turn cancerous, but said the teamhas made great efforts to remove potentially cancerous cells.

Hideyuki Okano, professor of molecular neurobiology at Keio University, who is researching the application of iPS-derived nerve cells to treat patients with spinal cord damage, said the risk was worth it.

Okano said the Osaka University's transplant, using tissues made from iPS cells from a donor, could be more effective than the existing therapy, which uses the patients own muscle tissues.

I understand that the transplanted tissues might become cancerous or cause an erratic heart rhythm, but the transplantation of the iPS-derived heart muscle tissues can be more effective than muscle tissue sheets made from the patients leg, Okano said.

Keio University is also planning to conduct clinical research using iPS-derived cells to regenerate heart tissues.

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Neuralstem (NASDAQ:CUR) & SpringWorks Therapeutics (NASDAQ:SWTX) Financial Review – Riverton Roll

By daniellenierenberg

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

Earnings & Valuation

This table compares Neuralstem and SpringWorks Therapeutics gross revenue, earnings per share and valuation.

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

Institutional and Insider Ownership

38.3% of Neuralstem shares are owned by institutional investors. Comparatively, 72.1% of SpringWorks Therapeutics shares are owned by institutional investors. 5.4% of Neuralstem shares are owned by insiders. Strong institutional ownership is an indication that hedge funds, endowments and large money managers believe a stock is poised for long-term growth.

Profitability

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

Analyst Ratings

This is a summary of current ratings and target prices for Neuralstem and SpringWorks Therapeutics, as reported by MarketBeat.

SpringWorks Therapeutics has a consensus price target of $35.50, suggesting a potential upside of 12.77%. Given SpringWorks Therapeutics higher possible upside, analysts plainly believe SpringWorks Therapeutics is more favorable than Neuralstem.

Summary

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

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.

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.

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Neuralstem (NASDAQ:CUR) & SpringWorks Therapeutics (NASDAQ:SWTX) Financial Review - Riverton Roll

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First pain treatment using human stem cells developed – THE WEEK

By daniellenierenberg

Scientists have developed the first treatment for pain using human stem cells, which provides lasting relief in mice in a single treatment, without side effects. If the treatment is successful in humans, it could be a major breakthrough in the development of new non-opioid, and non-addictive pain management, the researchers said.

"Nerve injury can lead to devastating neuropathic pain and for the majority of patients there are no effective therapies," said Greg Neely, an associate professor at the University of Sydney in Australia.

"This breakthrough means for some of these patients, we could make pain-killing transplants from their own cells, and the cells can then reverse the underlying cause of pain," Neely said in a statement.

The study, published in the journal Pain, used human induced pluripotent stem cells (iPSCs) derived from bone marrow to make pain-killing cells in the lab.

The iPSCs are cells which can develop into many different cell types in the body during early life, and growth.

The researchers then put the cells into the spinal cord of mice with serious neuropathic pain, caused by damage or disease affecting the nervous system.

"Remarkably, the stem-cell neurons promoted lasting pain relief without side effects," said study co-author Leslie Caron.

"It means transplant therapy could be an effective and long-lasting treatment for neuropathic pain. It is very exciting," Caron said.

Because the researchers can pick where to put the pain-killing neurons, they can target only the parts of the body that are in pain.

"This means our approach can have fewer side effects," said John Manion, a PhD student and lead author of research paper.

The stem cells used were derived from adult blood samples, the researchers noted.

Their next step will be to perform extensive safety tests in rodents and pigs.

They will then move to human patients suffering chronic pain within the next five years.

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Spinal injury researchers find a sweet spot for stem cell injections – New Atlas

By daniellenierenberg

As they do in many areas of medicine, stem cells hold great potential in treating injured spinal cords, but getting them where they need to go is a delicate undertaking. Scientists at the University of California San Diego (UCSD) are now reporting a breakthrough in this area, demonstrating a new injection technique in mice they say can deliver far larger doses of stem cells and avoid some of the dangers of current approaches.

The research focuses on the use of a type of stem cell known as a neural precursor cell, which can differentiate into different types of neural cells and hold great potential in repairing damaged spines. Currently, these are directly injected into the primary cord of nerve fibers called the spinal parenchyma.

As such, there is an inherent risk of (further) spinal tissue injury or intraparechymal bleeding, says Martin Marsala, professor in the Department of Anesthesiology at UCSD School of Medicine.

In experiments on rodents, Marsala and his team have demonstrated a safer and less invasive approach. The scientists instead injected the stem cells in between a protective layer around the spine called the pial membrane and the superficial layers of the spinal cord, a region known as the spinal subpial space.

This injection technique allows the delivery of high cell numbers from a single injection, says Marsala. 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.

Following these promising early results, the scientists are hopeful that stem cells injected in this way could one day greatly accelerate healing and improve the strength of cell-replacement therapies for several spinal neurodegenerative disorders, including spinal traumatic injury, amyotrophic lateral sclerosis and multiple sclerosis. But first will come experiments on larger animal models closer to the human anatomy in size, which will help them fine tune their technique for the best results.

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, says Marsala.

The research was published in the journal Stem Cells Translational Medicine.

Source: University of California San Diego

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What the Axolotl’s Limb-Regenerating Capabilities Have to Teach Us – Discover Magazine

By daniellenierenberg

As amphibians go, axolotls are pretty cute. These salamanders sport a Mona Lisa half-smile and red, frilly gills that make them look dressed up for a party. You might not want them at your soiree, though: Theyre also cannibals. While rare now in the wild, axolotls used to hatch en masse, and it was a salamander-eat-salamander world. In such a harsh nursery, they evolved or maybe kept the ability to regrow severed limbs.

Their regenerative powers are just incredible, says Joshua Currie, a biologist at the Lunenfeld-Tanenbaum Research Institute in Toronto whos been studying salamander regeneration since 2011. If an axolotl loses a limb, the appendage will grow back, at just the right size and orientation. Within weeks, the seam between old and new disappears completely.

And its not just legs: Axolotls can regenerate ovary and lung tissue, even parts of the brain and spinal cord.

The salamanders exceptional comeback from injury has been known for more than a century, and scientists have unraveled some of its secrets. It seals the amputation site with a special type of skin called wound epithelium, then builds a bit of tissue called the blastema, from which sprouts the new body part. But until recently, the fine details of the cells and molecules needed to create a leg from scratch have remained elusive.

With the recentsequencingandassemblyof the axolotls giant genome, though, and thedevelopment of techniques to modify the creatures genes in the lab,regeneration researchers are now poised to discover those details. In so doing, theyll likely identify salamander tricks that could be useful in human medicine

Already, studies are illuminating the cells involved, and defining the chemical ingredients needed. Perhaps, several decades from now, people, too, might regrow organs or limbs. In the nearer future, the findings suggest possible treatments for ways to promote wound-healing and treat blindness.

The idea of human regeneration has evolved from an if to a when in recent decades, says David Gardiner, a developmental biologist at the University of California, Irvine. Everybody now is assuming that its just a matter of time, he says. But, of course, theres still much to do.

In a working limb, cells and tissues are like the instruments in an orchestra: Each contributes actions, like musical notes, to create a symphony. Amputation results in cacophony, but salamanders can rap the conductors baton and reset the remaining tissue back to order and all the way back to the symphonys first movement, when they first grew a limb in the embryo.

The basic steps are known: When a limb is removed, be it by hungry sibling or curious experimenter, within minutes the axolotls blood will clot. Within hours, skin cells divide and crawl to cover the wound with a wound epidermis.

Next, cells from nearby tissues migrate to the amputation site, forming a blob of living matter. This blob, the blastema, is where all the magic happens, said Jessica Whited, a regenerative biologist at Harvard University, in a presentation in California last year. It forms a structure much like the developing embryos limb bud, from which limbs grow.

This movie shows immune cells, labeled to glow green, moving within a regenerating axolotl fingertip. Scientists know that immune cells such as macrophages are essential for regeneration: When they are removed, the process is blocked.

Finally, cells in the blastema turn into all the tissues needed for the new limb and settle down in the right pattern, forming a tiny but perfect limb. This limb then grows to full size. When all is done, you cant even tell where the amputation occurred in the first place, Whited tellsKnowable Magazine.

Scientists know many of the molecular instruments, and some of the notes, involved in this regeneration symphony. But its taken a great deal of work.

As Currie started as a new postdoc with Elly Tanaka, a developmental biologist at the Research Institute of Molecular Pathology in Vienna, he recalls wondering, Where do the cells for regeneration come from? Consider cartilage. Does it arise from the same cells as it does in the developing embryo, called chondrocytes, that are left over in the limb stump? Or does it come from some other source?

To learn more, Currie figured out a way to watch individual cells under the microscope right as regeneration took place. First, he used a genetic trick to randomly tag the cells he was studying in a salamander with a rainbow of colors. Then, to keep things simple, he sliced off just a fingertip from his subjects. Next, he searched for cells that stuck out say, an orange cell that ended up surrounded by a sea of other cells colored green, yellow and so on. He tracked those standout cells, along with their color-matched descendants, over the weeks of limb regeneration. His observations, reported in the journalDevelopmental Cellin 2016,illuminated several secrets to the regeneration process.

Regenerative biologist Joshua Currie labeled the cells in axolotls with a rainbow of colors, so that he could follow their migration after he amputated the tip of the salamanders fingertips. In this image, three days after amputation, the skin (uncolored) has already covered the wound. (Credit: Josh Currie)

For one thing, cell travel is key. Cells are really extricating themselves from where they are and crawling to the amputation plane to form this blastema, Currie says. The distance cells will journey depends on the size of the injury. To make a new fingertip, the salamanders drew on cells within about 0.2 millimeters of the injury. But in other experiments where the salamanders had to replace a wrist and hand, cells came from as far as half a millimeter away.

More strikingly, Currie discovered that contributions to the blastema were not what hed initially expected, and varied from tissue to tissue. There were a lot of surprises, he says.

Chondrocytes, so important for making cartilage in embryos, didnt migrate to the blastema (earlier in 2016, Gardiner and colleaguesreported similar findings). And certain cells entering the blastema pericytes, cells that encircle blood vessels were able to make more of themselves, but nothing else.

The real virtuosos in regeneration were cells in skin called fibroblasts and periskeletal cells, which normally surround bone. They seemed to rewind their development so they could form all kinds of tissues in the new fingertip, morphing into new chondrocytes and other cell types, too.

To Curries surprise, these source cells didnt arrive all at once. Those first on the scene became chondrocytes. Latecomers turned into the soft connective tissues that surround the skeleton.

How do the cells do it? Currie, Tanaka and collaborators looked at connective tissues further, examining the genes turned on and off by individual cells in a regenerating limb. In a 2018Sciencepaper, the team reported thatcells reorganized their gene activation profileto one almost identical, Tanaka says, to those in the limb bud of a developing embryo.

Muscle, meanwhile, has its own variation on the regeneration theme. Mature muscle, in both salamanders and people, contains stem cells called satellite cells. These create new cells as muscles grow or require repair. In a 2017 study inPNAS, Tanaka and colleagues showed (by tracking satellite cells that were made to glow red) that most, if not all, ofmuscle in new limbs comes from satellite cells.

If Currie and Tanaka are investigating the instruments of the regeneration symphony, Catherine McCusker is decoding the melody they play, in the form of chemicals that push the process along. A regenerative biologist at the University of Massachusetts Boston, she recently published arecipe of sorts for creating an axolotl limb from a wound site. By replacing two of three key requirements with a chemical cocktail, McCusker and her colleagues could force salamanders to grow a new arm from a small wound on the side of a limb, giving them an extra arm.

Using what they know about regeneration, researchers at the University of Massachusetts tricked upper-arm tissue into growing an extra arm (green) atop the natural one (red). (Credit: Kaylee Wells/McCusker Lab)

The first requirement for limb regeneration is the presence of a wound, and formation of wound epithelium. But a second, scientists knew, was a nerve that can grow into the injured area. Either the nerve itself, or cells that it talks to, manufacture chemicals needed to make connective tissue become immature again and form a blastema. In their 2019 study inDevelopmental Biology, McCusker and colleagues guided byearlier work by a Japanese team used two growth factors, called BMP and FGF, to fulfill that step in salamanders lacking a nerve in the right place.

The third requirement was for fibroblasts from opposite sides of a wound to find and touch each other. In a hand amputation, for example, cells from the left and right sides of the wrist might meet to correctly pattern and orient the new hand. McCusckers chemical replacement for this requirement was retinoic acid, which the body makes from vitamin A. The chemical plays a role in setting up patterning in embryos and has long been known to pattern tissues during regeneration.

In their experiment, McCuskers team removed a small square of skin from the upper arm of 38 salamanders. Two days later, once the skin had healed over, the researchers made a tiny slit in the skin and slipped in a gelatin bead soaked in FGF and BMP. Thanks to that cocktail, in 25 animals the tissue created a blastema no nerve necessary.

About a week later, the group injected the animals with retinoic acid. In concert with other signals coming from the surrounding tissue, it acted as a pattern generator, and seven of the axolotls sprouted new arms out of the wound site.

The recipe is far from perfected: Some salamanders grew one new arm, some grew two, and some grew three, all out of the same wound spot. McCusker suspects that the gelatin bead got in the way of cells that control the limbs pattern. The key actions produced by the initial injury and wound epithelium also remain mysterious.

Its interesting that you can overcome some of these blocks with relatively few growth factors, comments Randal Voss, a biologist at the University of Kentucky in Lexington. We still dont completely know what happens in the very first moments.

If we did know those early steps, humans might be able to create the regeneration symphony. People already possess many of the cellular instruments, capable of playing the notes. We use essentially the same genes, in different ways, says Ken Poss, a regeneration biologist at the Duke University Medical Center in Durham who describednew advances in regeneration, thanks to genetic tools, in the 2017Annual Review of Genetics.

Regeneration may have been an ability we lost, rather than something salamanders gained. Way back in our evolutionary past, the common ancestors of people and salamanders could have been regenerators, since at least one distant relative of modern-day salamanders could do it. Paleontologists have discovered fossils of300-million-year-old amphibians with limb deformities typically created by imperfect regeneration.Other members of the animal kingdom, such as certain worms, fish and starfish, can also regenerate but its not clear if they use the same symphony score, Whited says.

These fossils suggest that amphibians calledMicromelerpetonwere regenerating limbs 300 million years ago. Thats because the fossils show deformities, such as fused bones, that usually occur when regrowth doesnt work quite right. (Credit: Nadia B Frbisch et al./Proceedings of the Royal Society B, 2014)

Somewhere in their genomes, all animals have the ability, says James Monaghan, a regeneration biologist at Northeastern University in Boston. After all, he points out, all animals grow body parts as embryos. And in fact, people arent entirely inept at regeneration. We can regrow fingertips, muscle, liver tissue and, to a certain extent, skin.

But for larger structures like limbs, our regeneration music falls apart. Human bodies take days to form skin over an injury, and without the crucial wound epithelium, our hopes for regeneration are dashed before it even starts. Instead, we scab and scar.

Its pretty far off in the future that we would be able to grow an entire limb, says McCusker. I hope Im wrong, but thats my feeling.

She thinks that other medical applications could come much sooner, though such as ways to help burn victims. When surgeons perform skin grafts, they frequently transfer the top layers of skin, or use lab-grown skin tissue. But its often an imperfect replacement for what was lost.

Thats because skin varies across the body; just compare the skin on your palm to that on your calf or armpit. The tissues that help skin to match its body position, giving it features like sweat glands and hair as appropriate, lie deeper than many grafts. The replacement skin, then, might not be just like the old skin. But if scientists could create skin with better positional information, they could make the transferred skin a better fit for its new location.

Monaghan, for his part, is thinking about regenerating retinas for people who have macular degeneration or eye trauma. Axolotls can regrow their retinas (though, surprisingly, their ability to regenerate the lens is limited to hatchlings). He is working with Northeastern University chemical engineer Rebecca Carrier, whos been developing materials for use in transplantations. Her collaborators are testing transplants in pigs and people, but find most of the transplanted cells are dying. Perhaps some additional material could create a pro-regeneration environment, and perhaps axolotls could suggest some ingredients.

Carrier and Monaghan experimented with the transplanted pig cells in lab dishes, and found they were more likely to survive and develop into retinal cells if grown together with axolotl retinas. The special ingredientseems to be a distinct set of chemicals that exist on axolotl, but not pig, retinas.Carrier hopes to use this information to create a chemical cocktail to help transplants succeed. Even partially restoring vision would be beneficial, Monaghan notes.

Thanks to genetic sequencing and modern molecular biology, researchers can continue to unlock the many remaining mysteries of regeneration: How does the wound epithelium create a regeneration-promoting environment? What determines which cells migrate into a blastema, and which stay put? How does the salamander manage to grow a new limb of exactly the right size, no larger, no smaller? These secrets and more remain hidden behind that Mona Lisa smile at least for now.

10.1146/knowable-012920-1

This article originally appeared in Knowable Magazine, an independent journalistic endeavor from Annual Reviews.

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