CAMBRIDGE, Mass.--(BUSINESS WIRE)--QurAlis Corporation, a biotech company focused on developing precision therapeutics for amyotrophic lateral sclerosis (ALS) and other neurologic diseases, today announced the raise of a $42 million Series A financing, bringing the total funds raised to $50.5 million. The financing was led by LS Polaris Innovation Fund, lead seed investor Mission BioCapital, INKEF Capital and the Dementia Discovery Fund, and co-led by Droia Ventures. Additional new investors include Mitsui Global Investment and Dolby Family Ventures, joined by investments from existing investors Amgen Ventures, MP Healthcare Venture Management, and Sanford Biosciences. QurAlis intends to use this funding to support the development of new therapies for ALS and genetically related frontotemporal dementia (FTD), neurodegenerative diseases for which there is currently no cure.

This Series A funding will allow us to take the next major step in our growth and advance our lead programs into the clinic. Recent advances in science and technology have identified strong disease targets for specific groups of ALS and FTD patients. Combined with our proprietary human stem cell technologies and development capabilities, we believe we are placed in a very good position to bring forth real treatments, said Kasper Roet, Ph.D., Chief Executive Officer of QurAlis. The QurAlis team built this company from the ground up on a foundation of cutting-edge science and profound dedication to helping ALS patients above all else. The great support of our existing and new investors from the US, Europe and Japan underscores the international nature of our mission. We plan to use this funding to continue advancing ALS and FTD therapies for patients around the world who are in critical need of effective treatments.

As ALS can be caused by mutations in over 25 individual human genes, many of which also cause FTD, QurAlis strategy is to systematically investigate treatments targeting specific disease-causing mechanisms in patient sub-populations. The company evaluates a wide range of potential treatments through the companys transformative system that utilizes lab-grown neuronal networks derived from cells of ALS patients.

Between the companys strong scientific foundation and support by ALS luminaries Kevin Eggan and his co-founders, promising pipeline of potential ALS treatments, and its dedicated team of experts in the field of neurologic therapeutics, QurAlis is very well positioned to make a tremendous difference for patients with ALS and FTD, said Amy Schulman, Managing Partner of the LS Polaris Innovation Fund. We are proud to support their mission and have deep faith in their transformative technology, which has already supported the discovery of several promising ALS candidate therapeutics.

In connection with the financing round, Amy Schulman, Managing Partner of the LS Polaris Innovation Fund; Roel Bulthuis, Managing Partner at INKEF Capital; Jonathan Behr, Ph.D., Partner at the Dementia Discovery Fund; and Luc Dochez, Managing Partner at DROIA Ventures, will be joining Johannes Fruehauf, M.D., Ph.D., General Partner at Mission BioCapital, on QurAlis Board.

About ALS

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrigs disease, is a progressive neurodegenerative disease impacting nerve cells in the brain and spinal cord. ALS breaks down nerve cells, reducing muscle function and causing loss of muscle control. ALS can be traced to mutations in over 25 different genes and is often caused by a combination of multiple sub-forms of the condition. Its average life expectancy is three years, and there is currently no cure for the disease.

About QurAlis Corporation

QurAlis is developing precision therapeutics for ALS, a terminal disease that causes muscle paralysis through degeneration of the motor system. We are digging deep into the root causes of the multiple sub-forms of this destructive disease and focus our programs on tackling specific disease-causing mechanisms.

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QurAlis raises $42 Million Series A Financing to Develop New Therapies for Amyotrophic Lateral Sclerosis (ALS) - Business Wire

Spinal Cord Trauma Treatment Market: Global Industry Analysis 2012 2016 and Forecast 2017 2025is the recent report of Persistence Market Research that throws light on the overall market scenario during the period of eight years, i.e. 2017-2025. According to this report, Globalspinal cord trauma treatment marketis expected to witness significant growth during the forecast period.

This growth is expected to be primarily driven by increasing incidence of spinal cord trauma, and increasing government support to reduce the burden of spinal cord injuries. Additionally, development of nerve cells growth therapy is expected to boost the market in near future.

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Sales Revenue:Market Share, Growth Rate, Current Market Analysis.Product Revenue for Top Players: Market Share, Growth Rate, Current Market Situation Analysis.Industry Trends: United States and Other Regions Revenue, Status and Outlook.Market Segment: By Types, By Applications, By Regions/ Geography.Market Environment: Government Policies, Technological Changes, Market Risks.Market Drivers: Growing Demand, Reduction in Cost, Market Opportunities and Challenges.Competitive Landscape: By Manufacturers, Development Trends, Marketing Area

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The global market for spinal cord trauma treatment is is estimated to be valued atUS$ 2,276.3 Mnin terms of value by the end of 2017. The global spinal cord trauma treatment market is expected to expand at aCAGR of 3.7%over the forecast period to reach a value ofUS$ 3,036.2 Mnby 2025end.

Global Spinal Cord Trauma Treatment Market: Trends

Global Spinal Cord Trauma Treatment Market: Forecast by End User

On the basis of end user, the global spinal cord trauma treatment market is segmented into hospitals and trauma centers. Hospitals segment dominated the global spinal cord trauma treatment market in revenue terms in 2016 and is projected to continue to do so throughout the forecast period.

Hospitals and trauma centers segments are expected to approximately similar attractive index. Hospitals segment accounted for53.2%value share in 2017 and is projected to account for52.5%share by 2025 end.

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Proton Therapy Systems Market

Proton Therapy Market Segmented By Single Room, Multiple Room Set up Type with Head and Neck Cancer, Brain Cancer, Sarcoma Pediatric Cancer, Gastro-intestinal Cancer, Prostate Cancer, Lung Cancer Indication.For More Information

Trauma Fixation Devices Marketglobal trauma fixation devices market is estimated to represent more than US$ 450 Mn of the total market in 2017 and is estimated to reach little more than US$ 800 Mn by 2025 end, expanding at CAGR of 7.5% over the forecast period of 20172025.For More Information

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PMR : Spinal Cord Trauma Treatment Market Worth Will Reach US$ 3000 Mn According To Forecast By 2025 - Cole of Duty

Stem cells have long-held hope for many people with spinal cord injuries. Since their discovery in 1998, they have been used in thousands of studies to one day cure paralysis, but there is still no cure for those with chronic injuries. Despite this, scientists have come a long way, especially in recent years. You have likely heard about some of the research in the news, stirring more hope than ever before.

And the hope is not unfounded. The hard work and the millions of dollars going into this research is finally seeing results. Stem cells may not be the only key to finding a cure for a spinal cord injury, but they arent going anywhere and are being used in hundreds of studies around the world. Here are the ones you should know about.

The human body has millions of stem cells that can be found all over the body. Researchers at the Mayo Clinic were recently in the news for their results using fat-derived adult stem cells from the patients own body. This study recruited 10 individuals with traumatic spinal cord injuries. Each was injected with stem cells taken from the fat in their stomachs and was expanded in the lab for eight weeks. The injection was then in the lower lumbar area.

This treatment is brand new and has not been approved by the FDA, however, the study was granted special clearance. One individual in the study, a man in his fifties with an incomplete injury who had leg return and was able to walk slightly post-injury without treatment, saw a nearly 50% increase in his abilities after receiving the injection.

Researchers also made sure to wait until each person in the study had plateaued after their injuries to be sure that the results from the treatment were not results from the body still having a new injury. There is currently no further news on whether the FDA will approve this treatment for the general population.

Nearly a year ago, Japan's Health Ministry approved a trial that will involve four people with complete injuries. This stem cell trial uses induced pluripotent stem cells (IPS) taken from embryos and will be grown into two million nerve cells for each patient. These cells will then be injected into the injury site. This trial comes from Masaya Nakamura, a professor at Okano and Keio University, who saw improvements in animals after they underwent the procedure. There has that no updates on this trial since it was approved.

In Spain, a clinical trial has been underway for the last few years that uses stem cells taken from the patient's bone marrow and injected into their injury site. This research comes from Dr. Vaquero at the Puerta de Hierro University Hospital in Spain. His first trial in 2016 included people with complete injuries and his second trial in 2017 included people with incomplete injuries. Almost all patients who underwent the procedure saw some improvement, with some seeing more improvement than others. The trial is currently seeking 30 people with incomplete injuries for its next phase.

Dr. Steven Levy of MD Stem Cells is launching the SciExVR trial using a patient's stem cells from their bone marrow as well. This study is currently recruiting patients in the United States and will involve exoskeleton rehab as part of the trial. Learn more:http://mdstemcells.com/sciexvr

Dr. Wise Young, along with Rutgers University and his organization SCINetChina, has been approved for a study in the United States that will involve umbilical cord blood stem cells and oral lithium. This study will involve 27 people with complete chronic injuries levels C5-T11. You must be able to be in New Jersey for six months is chosen.

Six years ago in 2014, Dr. Raisman from Poland pioneered a study using nerve stem cells taken from the nose. These stem cells were taken from the olfactory bulb deep in the brain and were transplanted into the injury site along with nerve tissue taken from the patient ankle. This study is recruiting one person for the trial who has a perfectly severed spinal cord (by a knife or similar). The person recruited will also have to spend several years in Poland. To learn more, contacthttps://walk-again-project.org/#/en

Keep in mind that all the above stem cell trials are still trials and that they cannot promise any return of movement or sensation. It is always in your best interest to go into a trial with an open mind and to be hopeful, but be realistic at all times.

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The Latest In Stem Cell Therapy After SCI

Bioscience Americas and the Global Institute of Stem Cell Therapy and Research would like to extend a special thank you to the Christopher and Dana Reeve Foundation for their support relating to our work at the University California Irvin and the Anderson Laboratory. We have made exciting progress using stem cells to treat cervical spinal cord injuries because of their generosity.

Now, based on the results of Dr. Andersons Phase I/II clinical trial, our research partners are conducting a Phase II proof of concept trial using HuCNS-SC in cervical spinal cord injury. In this study, research participants are being treated between 10 to 23 months post-injury.

Spinal Cord Injury (SCI) is damage to the human spinal cord into three different segments of the neural tissue leading to a severe form of motor and sensory loss. The kind of damage can be differentiated as:

In most of the reported cases of SCI, damage can be due to trauma or disease. Apart from the physical damage and complete dependency on caregivers, SCI can be emotionally damaging as well. Due to dependency even on basic mobility, negative attitudes of suffering trauma forever and frequent mood swings can lead suffers to remove themselves from social participation. Thus more than 30% of the reported cases of SCI showed significant signs of depression and negative impact on the functional improvement of overall health.

How prevalent is SCI?

Since SCI is associated with the loss of mobility, paralysis, and mortality due to other opportunistic infections, it is known as one of the most critical and disastrous medical conditions. Every year around 2 million to 5 million people are reported to suffer from spinal cord injury. On an average, middle-aged and young adult males are more susceptible to SCI mainly due to avoidable causes such as road accidents, injury, falls or violence. Mortality associated with SCI has been observed to be the highest immediately after the injury than in later years. The risk of mortality doubles with the severity level and is observed to be strongly influenced by the immediate availability of the best medical care. Preventable secondary opportunistic infections are also reported to be a major cause of death in many SCI patients, especially in the lower income groups.

About 90% of patients in the age group of 20-45 have been reported to face other complications such as limited employment, decreased quality of life, and severe depression.

Factors responsible for SCI.

In general, a spinal cord injury is a result of to the severe damage to different parts of the spinal cord such as the vertebral column, ligaments or the spinal disks. This typically originates from sudden trauma to the spinal cord such as fracturing, crushing or dislocating one or more vertebrae. Additional damage has been reported due to excessive bleeding, swelling, inflammation as well as other opportunistic infections. The most common reported causes of Spinal Cord Injury are:

Symptoms Associated with SCI

In general, the severity, as well as the area of injury, are the factors to be of concern in most of the cases of Spinal Cord Injury. On the basis of severity of injury, SCI is classified as:

What goes wrong in Spinal Cord Injury?

The human spinal cord is a fragile bridge connecting the brain to the other organs of the body. The spinal cord is encased in a protective covering of spinal vertebrae of the spinal column to prevent its damage from shock or injury. Our central nervous system, i.e., brain and spinal cord, is made up of millions of cells which coordinate and communicate to pass on the information from the brain to the other organs of the body via the spinal cord. This information is passed in the form of electrical signals which are then decoded by the specific organ.

Each neuron is made up of a cellular body with a long slender projection called the nerve fiber. These fibers are attached to other fibers to form a dense network of cells. In general, neurons carrying messages down the cord from the brain to other organs of the body are known as Motor Neurons. These neurons control the muscles of some of the important internal organs of the body such as heart, stomach, intestine, etc. The neurons traveling up the cord to the brain are known as Sensory Neurons, carrying sensory information from skin, joints, and muscles to control our ability to sense, touch and regularize temperature.

These neurons are insulated from the outer side by the coating of Oligodendrocytes and myelin sheath. These cells insulate the neuron to protect them from sudden damage and shock.

If any of the above types of cells are affected due to sudden damage such as shearing, laceration, stretching or shock, then the network of cells is disturbed due to which the passage of information from the brain to the spinal cord and vice versa is halted.

How Stem Cells treatment can help.

Stem cells are the mother cells that are responsible for developing an entire human body from tiny two-celled embryos. Due to their unlimited divisions and strong power to differentiate into all the cells of different lineage, the power of stem cells has been harnessed by our technology to isolate them outside the human body, concentrate in a clean environment, and implant back.

Thus stem cells treatment involves administration of concentrated cells in the targeted area, wherein they can colonize in the damaged area, adapt the properties of resident stem cells and initiate some of the lost functions that have been compromised by the disease or injury.

Thus with our standardized, broad-based and holistic approach, it is now possible to obtain noticeable improvements in SCI cases, in the symptoms as well as their functional abilities.

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Spinal Cord Injury Recovery Through Stem Cell Therapy

They hold huge promise, but stem cell-based spinal cord treatments wont be clinically available in the near future

My three-year-old son was born with a very large spinal lipoma. He was considered quadriplegic. Through conventional physical and occupational therapies and surgery to remove some of the lipoma he has gained enough function to walk with a walker and use his arms. However, he is experiencing some regression as his nerves are dying.

I have saved the cord blood from his younger brother and sister. New research where mice are being paralyzed and then injected with stem cells looks very promising to us. The mice nerves that are sick or weak are being protected and strengthened. Our son needs his nerves protected from degeneration.

Conventional surgery is no longer an option because the nerve roots travel in and out of the lipoma and cannot be separated from the lipoma. Our only hope is to protect and strengthen what function he currently has.

My question is: How long before this type of stem cell therapy will be used on humans, more specifically children? And how do we get to be first in line? If it is 10 or 20 years away, there may be no way to save the function our son has worked so hard to gain. I havent read anything about risks or side effects. There have to be some, what are they? Also, are there other countries that are more aggressive in their use of stem cells on humans for treating paralysis resulting from spinal cord injury?

Barbara BourgeoisCentreville, Virginia, USA

There isnt an easy answer here, and Im not clear as to why function is being lost at this pointin particular, whether the lipoma is recurring. If this is the case, resolution of the lipoma is the main issue. In some instances, it is impossible to completely remove the tumor, severely limiting the potential benefits of secondary therapeutics (such as stem cells). However, on the topic of stem cells in particular, there are several issues to discuss.

First, there are many sources of stem cells, and this affects their potential clinical use. Cord blood-derived stem cells are probably the farthest away from potential clinical use for spinal cord injury at this point, because there has been less basic research done with them so far. Human embryonic and adult stem cell lines may be somewhat closer, but research on these in the laboratory has been somewhat mixedsome very promising results with regaining motor function, and some big potential concerns, such as causing tumor formation.

As a result, we are most likely still years away from testing these treatments in patients, even to establish safety. Some other kinds of cell treatments, such as ensheathing glial cells, are being tried in the clinic in China, Russia and Portugal based on previous laboratory research in the US. However, none of these overseas trials has been designed in accordance with US standards to rigorously test safety and efficacy, and it is very difficult to evaluate the patchy data coming out so far.

To sum up, as a researcher, I think stem cells hold a huge amount of promise, but we arent yet at a point where this work will be translated to the clinic in the immediate future.

Answered by Aileen J. Anderson ~ 1/22/2004

Posted on January 27th, 2004 in General SCI and Human Interest. Tagged: stem cells

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When Will Stem Cells Heal Spinal Cord Damage?

Akin to cystic fibrosis (CF), scientists understand that certain mutations contribute to the development of the fatal neurological disorder amyotrophic lateral sclerosis (ALS). And much like CF drugmaker Vertex, a small Cambridge, Massachusetts-based biotech is forging a path to engineering precision therapies to treat the disease that killed visionary physicist Stephen Hawking.

The company, christened QurAlis, now has $42 million in its coffers with three preclinical programs and 5 employees (including senior management) to combat an illness that has long flummoxed researchers, resulting in a couple of approved therapies over the course of decades, neither of which attacks the underlying cause of the rare progressive condition that attacks nerve cells located in the brain and spinal cord responsible for controlling voluntary muscles.

ALS garnered international attention when New York Yankees player Lou Gehrig abruptly retired from baseball in 1939, after being diagnosed with the disease. In 2014, ALS returned to the spotlight with the Ice Bucket Challenge, which involved people pouring ice-cold water over their heads, posting a video on social media, and donating funds for research on the condition.

QurAlis chief Kasper Roet, whose interest in ALS was piqued while he was working on his PhD at the Netherlands Institute for Neuroscience focusing on a treatment for spinal cord paralysis and moonlighting at the Netherlands Brain Bank as an ad-hoc autopsy team coordinator, saw an opportunity to combat ALS when Harvard scientists Kevin Eggan and Clifford Woolf pioneered some new stem cell technology.

Essentially, they found a way to take skin cells from a patient, turn them into stem cells, and turn those into the nerve cells that are degenerating. Thats the missing link, Roet said. So now we can finally use patients own cells to both do target discovery and develop potential therapeutics.

So Roet packed up his things and shifted base to Boston to learn more, with plans to head back to Europe to start a company. He never left. QurAlis was born in 2016, working out of a co-working space called LabCentral after winning a spot via an Amgen-sponsored innovation competition. The company was carved out of a collaboration with Eggans startup Q-State Biosciences, which developed laser technology to examine cell behavior examining how a neuron fires was imperative in the drug discovery process for ALS.

QurAlis, which counts Vertexs founding scientist Manuel Navia as an advisor, now has three preclinical programs. The furthest along is a therapy designed to target a specific potassium channel that is implicated in certain ALS patients the plan is to take that small molecule into the clinic next year, Roet said.

It has become really clear that if you understand why a specific tumor is developing you can develop very specific targeted therapies, he explained in an interview drawing a parallel between ALS and oncology. Thats exactly the same strategy that we are following for ALS. The genetics have shown that over 25 genes are causing the (ALS) mutations. Some of them work together, some of them are very dominant and work alone what we are doing is trying to get those specific proteins that are tied to very specific ALS populations, where we know that that specific target plays a very important and crucial role in the development of the disease.

In 2018, QurAlis scored seed funding from Amgen, Alexandria, and MP Healthcare Venture Management. The Series A injection was led by LS Polaris Innovation Fund, lead seed investor Mission BioCapital, INKEF Capital and the Dementia Discovery Fund, and co-led by Droia Ventures. Additional new investors include Mitsui Global Investment and Dolby Family Ventures, and existing investors Amgen Ventures, MP Healthcare Venture Management, and Sanford Biosciences also chipped in.

Roet is not sure how long these funds will last, particularly given the uncertainty of the coronavirus pandemic. But some of the capital will be used in hiring, given that the QurAlis team is comprised of a mere five people, including Roet.

Weve been very productive, he said. But we can definitely use some extra hands.

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Precision therapy approach secures small biotech $42M haul to combat disease that inspired the Ice Bucket Challenge - Endpoints News

Article In Brief

A mouse study shows that select transcription factors to the striatum can effectively and safely convert astrocytes to neurons to treat Huntington's disease.

Delivering two transcription factors to the striatum in a mouse model of Huntington's disease can safely convert astrocytes into neurons with high efficiency, according to a new study in the February 27 issue of Nature Communications.

The neurons grow to and wire up with their targets in the globus pallidus and substantia nigra, and remaining astrocytes proliferate to replace those that have been converted. The treatment extends the lifespan and improves the motor behavior of the mice.

What is exciting about this study is that the authors have clearly made cells that do what they are supposed to do, namely replace dying neurons in existing circuits, said Roger Barker, PhD, professor of clinical neuroscience and honorary consultant in neurology at the University of Cambridge and at Addenbrooke's Hospital, who was not involved in the work. I think the challenge of scaling up this strategy to the human Huntington's disease brain is pretty substantial, but nonetheless, this is an important discovery.

The new study, led by Gong Chen, PhD, builds on discoveries beginning in the mid-2000s showing that a small number of exogenously applied transcription factors could transform skin fibroblasts into stem cells, which could then be further converted to become virtually any cell type. That discovery was quickly followed by advances in direct reprogramming, in which one cell type is directly converted into another, skipping the stem cell intermediate.

Most of that work has taken place in vitro, and most attempts to use the strategy therapeutically have depended on transplantation of stem cells or newly converted cells.

We tried stem cell transplants to the mouse brain 10 years ago, but we couldn't find a lot of functional neurons, said Dr. Chen, professor at Guangdong-Hong Kong-Macau Institute of CNS Regeneration of Jinan University in Guangzhou, China.

It was also clear that anything you do in vitro, you eventually have to transplant, and that didn't seem to be a very promising technology, so I said, Let's try this in vivo, and put transcriptions factors directly into the mouse brain.

Dr. Chen initially tried introducing the transcription factor neurogenin 2, but the efficiency of conversion of astrocytes to neurons was very low, so he turned to the transcription factor NeuroD1, which Dr. Chen's group had previously shown could convert astrocytes into excitatory glutamatergic neurons.

In the current study, in order to generate GABAergic neurons, the team combined NeuroD1 with another transcription factor, D1x2, based on previous work showing its importance for generating GABAergic neurons.

The team packed the genes for the transcription factors into a recombinant adeno-associated virus vector (rAAV 2/5) and used an astrocyte-specific promoter to drive the transgene expression so that it preferentially expresses in astrocytes. They first injected the vector into the normal mouse striatum.

Surprisingly, this strategy worked very well at high efficiency, Dr. Chen said. After seven days, all transfected cells expressed astrocyte markers, indicating a high level of specificity in the vector. Of those cells, 81 percent co-expressed the two transcription factors. By 30 days, 73 percent of the cells expressing the transcription factors now expressed neuronal, rather than astrocytic markers, and were primarily GABAergic in character.

Next, Dr. Chen asked whether the remaining astrocytes could repopulate to replace those lost to conversion. Using immunostaining for astrocytes and neurons, as well as other techniques, the team found that the neuron/astrocyte ratio was unchanged, and that some remaining astrocytes could be found at different stages of cell division, suggesting the process facilitated astrocyte proliferation.

Dr. Chen then turned to the R6/2 mouse, the most common mouse model of Huntington's disease. He treated mice at 2 months of age, just as they began to show motor symptoms

As in the wild-type mice, astrocytes were converted to GABAergic neurons at high efficiency without altering the neuron/astrocyte ratio. The researchers observed similar results in a less-severe HD mouse model as well. Treated mice had only about half the degree of striatal atrophy as untreated mice. The converted neurons still contained aggregated huntingtin protein, but less than in native neurons, and similar to the reduced amount found in astrocytes in the mouse brain.

The real test of any cell therapy in neurodegenerative disease is whether the new cells can link into the existing circuits and provide functional benefit, feats that have been hard to achieve with transplanted fetal cells or stem cells.

Examining striatal slices from the treated mice, Dr. Chen found that the converted neurons displayed electrical properties largely identical to those of normal neurons, including resting potential, action potential threshold, firing amplitude, and firing frequency. They integrated into local circuits and behaved similarly to the native neurons around them. By tracking a marker contained in the AAV gene construct, they showed that converted neurons projected axons to the two basal ganglia targets of medium spiny neurons in the striatum, the globus pallidus and the substantia nigra.

Finally, Dr. Chen found that stride length and travel distance were both significantly improved in treated mice, though still falling below those of wild-type mice, and lifespan was significantly extended.

There were no hints of tumors in the mice, Dr. Chen noted. He suggested that in situ conversion is likely intrinsically safer in this regard than using stem cell-derived neurons, since a proliferative astrocyte is being converted into a non-proliferative neuron, with no residual pool of unconverted and potentially tumorigenic stem cells. We are actually reducing the tumor risk, he said.

Why the converted neurons developed appropriate neuronal connections is an important unanswered question, Dr. Chen said. He suggested there were two important factorsfirst, the astrocytes from which they arose are likely developmentally related to neighboring neurons, and thus may express similar position markers that help guide them to the right targets, just like the native neurons. Second, those remaining neurons may also provide guide tracks for the newly growing axons.

This conversion technique is not limited to Huntington's disease, he stressed, noting that his team last year published a paper showing promise in ischemic stroke, and work is underway to test its potential in Alzheimer's disease, Parkinson's disease, spinal cord injury, and ALS. He is also moving on to testing in non-human primates, setting the stage for eventual human trials.

I think eventually we will want to correct the Huntington's mutation as well, Dr. Chen said, for instance by using CRISPR, but he pointed out that while that strategy can repair diseased neurons, it cannot make new ones, like astrocyte-to-neuron conversion can.

This study is really elegantly done, commented Veronica Garcia, PhD, who has studied astrocytes derived from induced pluripotent stem cells from Huntington's disease patients as a postdoctoral scientist working with Clive Svendsen, PhD, in the Regenerative Medicine Institute at Cedars-Sinai Medical Center in Los Angeles.

The conversion efficiency is similar between wild-type and disease models, suggesting that the disease process is not interfering with the conversion, she said.

Astrocyte depletion does not seem to be a problem, at least in the short term, but Dr. Garcia noted there is a limit on the number of divisions astrocytes appear able to undergo, after which they lose the ability to proliferate. That may be a problem for chronic treatment, she suggested. Nonetheless, these results really look promising for therapeutic development.

The concept of trying to reprogram cells in situ to take on the phenotype of the cells that are lost is not new, commented Dr. Barker, but being able to do it with any degree of efficiency, to make enough cells to make a significant difference, has been problematic. For that reason, and because the cells grow to their target sites and make connections, these results are surprising.

A major hurdle for clinical trials, he noted, will be scaling up to the human striatum, which has approximately 100 times the volume of that in the mouse. Delivering the vector to such a large volume will be a significant challenge, he said, along with determining whether this approach will really work in a disease that affects many different brain structures such as in HD.

Dr. Chen is co-founder of NeuExcell Therapeutics Inc, which will develop clinical trials in the future. Drs. Barker and Garcia disclosed no conflicts.

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Researchers Convert Astrocytes to Neurons In Vivo to Treat... : Neurology Today - LWW Journals

Spinal Cord Trauma Treatment Market: Global Industry Analysis 2012 2016 and Forecast 2017 2025is the recent report of Persistence Market Research that throws light on the overall market scenario during the period of eight years, i.e. 2017-2025. According to this report, Globalspinal cord trauma treatment marketis expected to witness significant growth during the forecast period.

This growth is expected to be primarily driven by increasing incidence of spinal cord trauma, and increasing government support to reduce the burden of spinal cord injuries. Additionally, development of nerve cells growth therapy is expected to boost the market in near future.

What the report encloses for the readers:

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Company Profiles

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The global market for spinal cord trauma treatment is is estimated to be valued at US$ 2,276.3 Mn in terms of value by the end of 2017. The global spinal cord trauma treatment market is expected to expand at a CAGR of 3.7% over the forecast period to reach a value of US$ 3,036.2 Mn by 2025end.

Global Spinal Cord Trauma Treatment Market: Trends

Global Spinal Cord Trauma Treatment Market: Forecast by End User

On the basis of end user, the global spinal cord trauma treatment market is segmented into hospitals and trauma centers. Hospitals segment dominated the global spinal cord trauma treatment market in revenue terms in 2016 and is projected to continue to do so throughout the forecast period.

Hospitals and trauma centers segments are expected to approximately similar attractive index. Hospitals segment accounted for 53.2% value share in 2017 and is projected to account for 52.5% share by 2025 end.

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Global Spinal Cord Trauma Treatment Market: Forecast by Injury Type

On the basis of injury type, the global spinal cord trauma treatment market is segmented into complete spinal cord injuries and partial spinal cord injuries.

Partial spinal cord trauma treatment segment is expected to show better growth than the completed spinal cord treatment segment due to higher growth in the incidence rate of partial spinal cord trauma than the complete spinal cord trauma. With US$ 1,870.3 Mn market value in 2025, this segment is likely to expand at CAGR 3.8% throughout the projected period.

Global Spinal Cord Trauma Treatment Market: Forecast by Treatment Type

On the basis of treatment type, the global spinal cord trauma treatment market is segmented into corticosteroid, surgery, and spinal traction segments.

Surgery segment dominated the global spinal cord trauma treatment market in revenue terms in 2016 and is projected to continue to do so throughout the forecast period. Surgery segment is the most attractive segment, with attractiveness index of 2.6 over the forecast period.

Global Spinal Cord Trauma Treatment Market: Forecast by Region

This market is segmented into five regions such as North America, Latin America, Europe, APAC and MEA. Asia-Pacific account for the largest market share in the global spinal cord trauma treatment market.

Large patient population due to the high rate of road accidents and crime is making the Asia Pacific region most attractive market for spinal cord trauma treatment. On the other hand, MEA and Latin America is expected to be the least attractive market for spinal cord trauma treatment, with attractiveness index of 0.3 and 0.5 respectively over the forecast period.

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Spinal Cord Trauma Treatment Market to Register CAGR 3.7% Growth in Revenue During the Forecast Period 2025 - Jewish Life News

Researchers at Temple University have made a discovery that raises hope of restored functions for people with spinal cord injuries and other related issues.

In a study published in Molecular Therapy, researchers said they were able to regenerate neurons in mice that had spinal cord injury and optic nerve damage. They succeeded in restoring lost functions in the animals with the aid of a molecule called Lin28.

Several thousands of people suffer permanent losses of motor function and sensation due to spinal cord injury and similar conditions every year. These are results of severe damage or separation of axons.

Humans can regenerate most of the tissues in their bodies when damaged. Axons, however, fall among key body components that they are unable to redevelop when damaged.

Read Also: University of Freiburg Identifies the Neurons Responsible for Rapid Eye Movements During Sleep

The new research by the Temple University scientists interestingly shows that Lin28 helps to mend axons. With the aid of the molecule, mice showed gains in sensation and recovery of motor function.

Our findings show that Lin28 is a major regulator of axon regeneration and a promising therapeutic target for central nervous injuries, said lead researcher Dr. Shuxin Li, MD, PhD.

The regenerative capacity of Lin28 was observed when it was expressed at levels higher than normal.

It was the first time scientists showed the potential of the molecule to help repair spinal cord injuries.

Axons are nerve fibers that project from neurons. They form networks that help to pass signals from the brain to different parts of the body. These long nerve fibers literally serve as communication cables.

The brain, for instance, depends on axons to be able to communicate with muscles. It is this interaction that enables movement in response to stimuli.

When axons are severed as a result of an injury or accident, sensation and motor function losses result. These changes last for the rest of a persons life since the structures do not regenerate.

In the current research, scientists decided to explore the ability of Lin28 to regrow neurons. They developed an interest in this because of how it determines whether or not stem cells differentiate.

The researchers developed a model involving over-expression of the focal molecule in certain tissues in mice. They put the animals in different groups containing those having a spinal cord injury or optic nerve damage after becoming adults.

Also, the team had a different group of mice with normal levels of Lin28 expression as well as spinal cord or optic nerve injuries. To examine tissue repair effects, the animals were injected with a viral vector that increases the expression of the molecule.

Expressions of Lin28 above normal levels promoted regeneration of axons in all the animals.

Lin28 injections led to axons extending up to 3 mm away from the point of severance or damage in animals with spinal cord injury. Axons grew again all along the whole length of the optic nerve tract.

The use of molecule injections proved to be the most effective means of restoring damaged axons.

When the researchers assessed the walking and sensory capabilities of the animals, they found that Lin28 therapy led to great improvements.

At the moment, there is no restorative treatment for people with injuries involving the spinal cord or optic nerve tracts, which link to the retina.

Li said the level of axon regeneration seen in the study could be highly significant in clinical terms. The professor of anatomy and cell biology revealed that one of his immediate plans was finding a means of making Lin28 helpful to human patients.

Read Also: UC Berkeley Researchers Restore Vision in Mice Through Gene Insertion

He and colleagues need to create a carrier (or vector) for the molecule that would improve its expression when injected. They need to find a means of precisely targeting damaged axons with the treatment.

The researchers also planned to learn more about the Lin28 signaling pathway. Li expressed a suspicion that the molecule possibly uses more than one pathway to promote repair.

To support growth, the molecule seems to work with several others that could potentially be combined with it for more effective therapy.

References

https://www.cell.com/molecular-therapy-family/molecular-therapy/fulltext/S1525-0016(20)30191-X

See the article here:
Temple University: Spinal Cord Injury and Optic Nerve Damage Repaired With Growth-Regulating Molecule - Gilmore Health News

DetailsCategory: DNA RNA and CellsPublished on Wednesday, 06 May 2020 18:08Hits: 268

CARLSBAD, CA, USA I May 06, 2020 ILineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs, today announced 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 (AMD), were published online via the ARVOLearn platform as part of the 2020 Association for Research in Vision and Ophthalmology (ARVO) Meeting. The 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 (Abstract # 3363764), was presented by Christopher D. Riemann, M.D., Vitreoretinal Surgeon and Fellowship Director, Cincinnati Eye Institute (CEI) and University of Cincinnati School of Medicine. Dr. Riemanns presentation is available on the Media page of the Lineage website. Lineage will also host a live call with Dr. Riemann, on Monday, May 11, 2020 at 5:00 p.m. ET/2:00 p.m. PT to further discuss the results of treatment with OpRegen. Interested parties can access the call on the Events and Presentations section of Lineages website.

This update is significant as it builds on our earlier reports of gains in visual acuity and provides a more comprehensive picture of treatment with OpRegen for dry AMD, with meaningful improvements in the progression of geographic atrophy, visual acuity, and reading speed observed in our first Cohort 4 patient and first Orbit SDS with thaw-and-inject formulation dosed patient, stated Brian M. Culley, Lineage CEO. As dry AMD is a slow and progressive disease, it takes many months to observe changes to retinal anatomy or visual acuity. With the benefit of longer follow-up, we now can report that some OpRegen treated patients are able to see better, have less growth in their area of GA, and are able to read faster, all of which represent significant enhancements to vision and quality of life metrics. In addition to these individual results, the pooled data continues to suggest a treatment effect in both visual acuity and GA progression. Notably, we also are reporting additional evidence that OpRegen cells remain present for at least 4 years and hope that longer follow-up periods will reinforce a growing body of evidence that OpRegen is well-tolerated and can provide sustained and clinically meaningful benefits with a single dose of RPE cells. Our near-term objective is to treat and monitor the final four patients in Cohort 4 of the current study and utilize these data to direct our clinical, regulatory, and partnership discussions. Our goal is to combine the best cell line, the best production process, and the best delivery system, to position OpRegen as the front-runner in the race to address the unmet need in the potential billion-dollar dry AMD market.

As a principal investigator on the OpRegen clinical study, I am excited to present this most recent update, where all Cohort 4 patients treated with OpRegen had improved Best Corrected Visual Acuity up to one year or at their last visit, demonstrating a substantial treatment response, stated Christopher D. Riemann, M.D. The pooled Cohort 4 data demonstrate a significant, greater than 10-letter sustained visual acuity improvement over the entire followup period. Reading center assessments of GA also suggest a reduction in GA progression in the OpRegen treated eye when compared to fellow eye in Cohort 4. I am encouraged by the results observed in patients treated to date with OpRegen and I look forward to dosing patients in this study at CEI.

KOL Call Information and Webcast

Lineage will host a conference call with Dr. Riemann, on Monday, May 11, 2020 at 5:00 p.m. ET/2:00 p.m. PT to further discuss the results following treatment with OpRegen. A live webcast of the conference call will be available online in the Events and Presentations section of Lineages website. Interested parties may also access the conference call by dialing (866) 888-8633 from the U.S. and Canada and (636) 812-6629 from elsewhere outside the U.S. and Canada and should request the Lineage Cell Therapeutics Call. A replay of the webcast will be available on Lineages website for 30 days and a telephone replay will be available through May 19, 2020, by dialing (855) 859-2056 from the U.S. and Canada and (404) 537-3406 from elsewhere outside the U.S. and Canada and entering conference ID number 6597936.

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 to either 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 three allogeneic (off-the-shelf) product candidates: (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase 1/2a 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 1/2a development for the treatment of acute spinal cord injuries; and (iii) VAC2, a cancer immunotherapy of antigen-presenting dendritic cells in Phase 1 development for the treatment of non-small cell lung cancer. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

SOURCE: Lineage Cell Therapeutics

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Lineage Cell Therapeutics Reports New Data With OpRegen for the Treatment of Dry AMD With Geographic Atrophy | DNA RNA and Cells | News Channels -...

When the axons that extend from neurons break during a spinal cord injury, the result is often a lifelong loss of motor functioning, because vital connections from the brain to other body parts cannot be restored. Now, researchers from Temple Universitys Lewis Katz School of Medicine say they may have found a way to recover some functions lost to axon breaks.

The researchers discovered that boosting levels of a protein called Lin28 in injured spinal cords of mice prompts the regrowth of axons and repairs communication between the brain and body. Lin28 also helped repair injured optic nerves in the animals, they reported in the journal Molecular Therapy.

The Temple team zeroed in on Lin28 because its a known regulator of stem cells, meaning it controls their ability to differentiate into various cells in the body. The researchers examined the effects of Lin28 on spinal cord and optic nerve injuries using two mouse models: one that was engineered to express extra Lin28 and another that was normal and was given the protein after injury via a viral vector.

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All of the mice experienced axon regeneration, the researchers reported. But they found that the best results occurred in the normal mice that received Lin28 injections post-injury. In fact, in animals with optic nerve injuries, the axons regrew to the point where they filled the entire tract of the nerve.

Lin28 treatment after injury improved coordination and sensation in the mice, the researchers reported.

"We observed a lot of axon regrowth, which could be very significant clinically, since there currently are no regenerative treatments for spinal cord injury or optic nerve injury," said senior author Shuxin Li, M.D., Ph.D., professor of anatomy and cell biology at the Lewis Katz School of Medicine, in a statement.

RELATED: Gene therapy with 'off switch' restores hand movement in rats with spinal cord injury

Lin28 is already a target of interest, though it has garnered the most attention so far in cancer research. Startup Twentyeight-Seven Therapeutics is developing a small molecule that inhibits the protein in the hopes that doing so will boost Let-7, a cancer-suppressing microRNA. The company raised more than $82 million in a series A financing last year.

Several new approaches for repairing spinal cord injuries are under investigation, most notably gene therapy. King's College researchers are working on a gene therapy that repairs axons by prompting the production of the enzyme chondroitinase. A UT Southwestern team is targeting the gene LZK to increase levels of supportive nervous system cells called astrocytes in response to spinal injuries.

The Temple team has a two-pronged approach to further developing their Lin28-directed treatment. They hope to develop a vector that can be safely delivered by injection and that would deliver the therapy directly to damaged neurons. They also plan to study other molecules in the Lin28 signaling pathway.

"Lin28 associates closely with other growth signaling molecules, and we suspect it uses multiple pathways to regulate cell growth," Li said, potentially revealing other therapeutic molecules that could further boost neuron repair.

Link:
Repairing spinal cord injuries with a protein that regulates axon regeneration - FierceBiotech

Spinal Cord Trauma Treatment Market: Global Industry Analysis 2012 2016 and Forecast 2017 2025is the recent report of Persistence Market Research that throws light on the overall market scenario during the period of eight years, i.e. 2017-2025. According to this report, Globalspinal cord trauma treatment marketis expected to witness significant growth during the forecast period.

This growth is expected to be primarily driven by increasing incidence of spinal cord trauma, and increasing government support to reduce the burden of spinal cord injuries. Additionally, development of nerve cells growth therapy is expected to boost the market in near future.

Report Highlights:

Get Sample Copy of Report @ https://www.persistencemarketresearch.com/samples/17353

Company Profiles

Get To Know Methodology of Report @ https://www.persistencemarketresearch.com/methodology/17353

The global market for spinal cord trauma treatment is is estimated to be valued at US$ 2,276.3 Mn in terms of value by the end of 2017. The global spinal cord trauma treatment market is expected to expand at a CAGR of 3.7% over the forecast period to reach a value of US$ 3,036.2 Mn by 2025end.

Global Spinal Cord Trauma Treatment Market: Trends

Global Spinal Cord Trauma Treatment Market: Forecast by End User

On the basis of end user, the global spinal cord trauma treatment market is segmented into hospitals and trauma centers. Hospitals segment dominated the global spinal cord trauma treatment market in revenue terms in 2016 and is projected to continue to do so throughout the forecast period.

Hospitals and trauma centers segments are expected to approximately similar attractive index. Hospitals segment accounted for 53.2% value share in 2017 and is projected to account for 52.5% share by 2025 end.

Access Full Report @ https://www.persistencemarketresearch.com/checkout/17353

Global Spinal Cord Trauma Treatment Market: Forecast by Injury Type

On the basis of injury type, the global spinal cord trauma treatment market is segmented into complete spinal cord injuries and partial spinal cord injuries.

Partial spinal cord trauma treatment segment is expected to show better growth than the completed spinal cord treatment segment due to higher growth in the incidence rate of partial spinal cord trauma than the complete spinal cord trauma. With US$ 1,870.3 Mn market value in 2025, this segment is likely to expand at CAGR 3.8% throughout the projected period.

Global Spinal Cord Trauma Treatment Market: Forecast by Treatment Type

On the basis of treatment type, the global spinal cord trauma treatment market is segmented into corticosteroid, surgery, and spinal traction segments.

Surgery segment dominated the global spinal cord trauma treatment market in revenue terms in 2016 and is projected to continue to do so throughout the forecast period. Surgery segment is the most attractive segment, with attractiveness index of 2.6 over the forecast period.

Global Spinal Cord Trauma Treatment Market: Forecast by Region

This market is segmented into five regions such as North America, Latin America, Europe, APAC and MEA. Asia-Pacific account for the largest market share in the global spinal cord trauma treatment market.

Large patient population due to the high rate of road accidents and crime is making the Asia Pacific region most attractive market for spinal cord trauma treatment. On the other hand, MEA and Latin America is expected to be the least attractive market for spinal cord trauma treatment, with attractiveness index of 0.3 and 0.5 respectively over the forecast period.

Read more:
Adoption of Spinal Cord Trauma Treatment Market to Increase During the COVID-19 Period on back of Increased Consumer Demand - Jewish Life News

Astrocytes play a direct role in the regulation of neuronal circuits involved in learning and memory, according to new research from Baylor College of Medicine and M.D. Anderson Cancer Center.

Huang et al reveal region-specific transcriptional dependencies for astrocytes and identify astrocytic NFIA as a key transcriptional regulator of hippocampal circuits. Image credit: Huang et al, doi: 10.1016/j.neuron.2020.03.025.

Astrocytes are star-shaped glial cells in the brain and spinal cord.

They have unique cellular, molecular and functional properties and outnumber neurons by over fivefold. They occupy distinct brain regions, indicating regional specialization.

There is evidence suggesting that transcription factors proteins involved in controlling gene expression regulate astrocyte diversity.

A team led by Professor Benjamin Deneen from Baylor College of Medicine looked to get a better understanding of the role transcription factor NFIA, a known regulator of astrocyte development, played in adult mouse brain functions.

The researchers worked with a mouse model they had genetically engineered to lack the NFIA gene specifically in adult astrocytes in the entire brain.

They analyzed several brain regions, looking for alterations in astrocyte morphology, physiology and gene expression signatures.

We found that NFIA-deficient astrocytes presented defective shapes and altered functions, Professor Deneen said.

Surprisingly, although the NFIA gene was eliminated in all brain regions, only the astrocytes in the hippocampus were severely altered. Other regions, such as the cortex and the brain stem, were not affected.

Astrocytes in the hippocampus also had less calcium activity calcium is an indicator of astrocyte function as well as a reduced ability to detect neurotransmitters released from neurons.

NFIA-deficient astrocytes also were not as closely associated with neurons as normal astrocytes.

Importantly, all these morphological and functional alterations were linked to defects in the animals ability to learn and remember, providing the first evidence that astrocytes are to some extent controlling the neuronal circuits that mediate learning and memory.

Astrocytes in the brain are physically close to and communicate with neurons. Neurons release molecules that astrocytes can detect and respond to, Professor Deneen said.

We propose that NFIA-deficient astrocytes are not able to listen to neurons as well as normal astrocytes, and, therefore, they cannot respond appropriately by providing the support needed for efficient memory circuit function and neuronal transmission. Consequently, the circuit is disrupted, leading to impaired learning and memory.

The findings were published online in the journal Neuron.

_____

Anna Yu-Szu Huang et al. Region-Specific Transcriptional Control of Astrocyte Function Oversees Local Circuit Activities. Neuron, published online April 21, 2020; doi: 10.1016/j.neuron.2020.03.025

View post:
Study Reveals New Role of Astrocytes in Brain Function | Neuroscience - Sci-News.com

This phase I clinical study is an open clinical trial to investigate the safety of the intrathecal application of Neuro-Cells in the treatment of end stage (chronic), traumatic complete (AIS grade A) and incomplete (AIS grade B/C) SCI patients. To that purpose, after inclusion in this study >1 year and less than 5 years after their SCI-event, 10 patients will be included. All patients are invited to visit the trial hospital every month during this 3-months study for appreciation of their possible (S)AEs and/or SUSARs, for physical examination and a biochemical analysis of their blood/urine. Day 0 and day 90 they also undergo a comprehensive neurological examination, the AISIAms, ASIAss and Pain perception.

Finally, the participants are also invited to undergo neurological examinations at day 360 and 720. The purpose of this neurological assessment is to explore in patients if a late administration of Neuro-Cells may have some beneficial effects on the neurological condition of the chronic SCI patient.

All patients undergo a BM harvesting at the start of their participation in the study and will undergo one LP, performed to administer Neuro-Cells. The study is open and descriptive, and no randomization takes place. All patients are followed up until approximately 3 months after the time of administration. After these 3 months, the safety part of this study ends. Patients are invited for a neurological assessment 9 months later (day 360) to explore if Neuro-Cells may have a beneficial effect when given to end stage patients with a traumatic SCI.

The safety part of the study is completed when the last patient finishes his/her visit at day 90. The explorative part of the study ends approximately one year after the time of inclusion at day 720.

The rest is here:
Safety Stem Cells in Spinal Cord Injury - Full Text View ...

I awoke on Monday morning to the sad news that Max Randell had passed away on April 18. He would have been 23 on October 9.

Maxie wasnt expected to live past the age of 8, or even much past toddlerhood, according to some doctors. But gene therapy, and his incredible family, had something to say about that. COVID-19 didnt claim him his body just tired of fighting.

Max Randells legacy is one of hope, to the rare disease community whose family members step up to participate in the clinical trials that lead to treatments. In this time of the pandemic, attention has, understandably, turned somewhat away from the many people who live with medical limitations all the time. Ill explore that story next week.

A Devastating Diagnosis

Max was diagnosed at 4 months of age with Canavan disease, an inherited neuromuscular disease that never touched his mind nor his ability to communicate with his eyes, even though his body increasingly limited what he could do. Fewer than a thousand people in the US have the condition.

Canavan disease is an enzyme deficiency that melts away the myelin that insulates brain neurons. Gene therapy provides working copies of the affected gene, ASPA.

Babies with Canavan disease are limp and listless. Most never speak, walk, or even turn over. Yet their facial expressions and responses indicate an uncanny awareness. A child laughs when his dad makes a fart-like noise; a little girl flutters her fingers as if they are on a keyboard when a friend plays piano. Theyre smart.

Today, with excellent speech, occupational, and physical therapy and earlier diagnosis, people with Canavan disease can live into their teens or twenties. Those with mild mutations live even longer.

Maxs passing is a tragedy, but he taught researchers about gene therapy to the brain. And that may help others.

Gene Therapy for Canavan

Max had his first gene therapy at 11 months of age and a second a few years later, after slight backsliding when clinical trials halted in the wake of the death of Jesse Gelsingerin a gene therapy trial for a different disease.

Ive written about Maxs journey through many editions of my human genetics textbook, in my book ongene therapy, and in several DNA Science posts, listed at the end.

Ive had the honor to attend two of Maxs birthday parties, which celebrate Canavan kids and the organization that his family founded, Canavan Research Illinois. At one party I brought along birthday cards that students whod read my gene therapy book made for him. And his grandma Peggy, who emailed me of his passing this past Monday, showed me how Max communicated with eyeblinks of differing duration and direction.

Heres what his mom Ilyce wrote about one yearly gathering:

This year will be the 20th Annual Canavan Charity Ball. Each year as I plan this event Im faced with the undeniable reality that theres a chance Maxie wont be here by the time the day rolls around. With each passing year this fear grows stronger and it becomes increasingly difficult to put into print that our annual event is in honor of Maxies birthday. Ive been talking to Maxie a lot lately about his life. He feels happy, strong, loved, content, productive, and fulfilled and he is looking forward to his upcoming 21st birthday. Im excited to celebrate this incredible milestone.

Maxs parents and brother Alex have had the unusual experience of time, of being able to watch their loved one as the years unfolded following gene therapy. They were able to see more subtle improvements than can the parents whose children have more recently had gene therapy to treat a brain disease. Parents watch and wait and hope that language will return, or that a child will become more mobile or less hyperactive, depending on the treated condition. The changes may be subtle, or slow, or restricted and thats what Max taught the world.

For him, the viruses that ferried the healing genes into his brain seem to have gathered at his visual system. His parents noticed improvements in the short term, just before his first birthday, as well as long term.

Within two to three weeks, he started tracking with his eyes, and he got glasses. He became more verbal and his motor skills improved. His vision is still so good that his ophthalmologist only sees him once a year, like any other kid with glasses. She calls him Miracle Max, Ilyce told me in 2010.

In 2016 I heard from Ilyce again:

I wanted to give you an update on Maxie. Hes going to be 19 on October 9th. He graduated from high school in June and is beginning a work program on Monday. Its been very exciting to watch him grow into a young man!

Max had an appointment with his ophthalmologist this week and his vision continues to improve. His doctor said that the gene is still active in his brain because his optic nerve shows absolutely no signs of degeneration and looks the same each year. I wish we could have been able to express the gene throughout more of his brain, but I am grateful for the treatments because of the progress hes made.

Even though gene therapy wasnt a cure for Max, the things we are experiencing definitely give me a lot of hope that once the delivery system is perfected, I can see a potential cure for Canavan disease in the future. Just knowing that the gene is still there 15 years later gives me confidence that a one-time gene transfer would actually work!

Maxs gene therapy circa 2002 targeted less than 1% of brain cells, with fewer viral vectors than are used to deliver healing genes in todays clinical trials. But it looks like some of the vectors may have made their way beyond the optic nerves, judging by the interest in math he had in high school and his critical thinking skills.

A Choice of Gene-Based Therapies

When the Randell family decided to pursue gene therapy, it was pretty much the only game in town. Thats changed.

Only two gene therapies have been approvedin the U.S. But a search at clinicaltrials.gov yielded 602 entriesdeploying the technology. The list still rounds up the usual suspects of years past mostly immune deficiencies, eye disorders, or blood conditions, with a few inborn errors of metabolism.

But one clinical trial mentions the gene-editing tool CRISPR, which can replace a mutant gene, not just add working copies as classical gene therapy does. TheCRISPRtrial is an experiment on stem cells removed from patients with Kabuki syndrome, which affects many body systems.

Spinal muscular atrophy now has two FDA-approved treatments, one an antisense therapy (Spinraza) that silences a mutation and the other (Zolgensma) a gene therapy that infuses copies of the functioning gene. Without treatment, the destruction of motor neurons in the spinal cord is usually lethal by age two.

In 2018, FDA approved the first drug based on RNA interference (RNAi), yet another biotechnology. It silences gene expression, which is at the RNA rather than the DNA level of the other approaches. Onpattro treats the tingling, tickling, and burning sensations from the rare condition hereditary transthyretin-mediated amyloidosis.

When I wrote my book on gene therapy in 2012, the technology was pretty much the only choice of research to pursue besides protein-based therapies like enzyme replacement. Now families raising funds for treatments for single-gene diseases can add antisense, RNAi, and CRISPR gene editing to the list of possibilities.

In any battle, a diversity of weapons ups the odds of defeating the enemy.

RIP Max Randell.

DNA Science posts:

Fighting Canavan: Honoring Rare Disease Week

A Brothers Love Fights Genetic Disease

Gene Therapy for Canavan Disease: Maxs Story

Celebrating the Moms of Gene Therapy

To support research:Canavan Research Illinois

See the original post:
A Tribute to Max Randell, Gene Therapy Pioneer - PLoS Blogs

(MENAFN - iCrowdNewsWire) Apr 23, 2020

' Spinal Cord Trauma Treatment Market: Global Industry Analysis 2012 2016 and Forecast 2017 2025' is the recent report of Persistence Market Research that throws light on the overall market scenario during the period of eight years, i.e. 2017-2025. According to this report, Global spinal cord trauma treatment market is expected to witness significant growth during the forecast period.

This growth is expected to be primarily driven by increasing incidence of spinal cord trauma, and increasing government support to reduce the burden of spinal cord injuries. Additionally, development of nerve cells growth therapy is expected to boost the market in near future.

Get To Know Methodology of Report @ https://www.persistencemarketresearch.com/methodology/17353

Company Profiles

Get Sample Copy of Report @ https://www.persistencemarketresearch.com/samples/17353

The global market for spinal cord trauma treatment is is estimated to be valued at US$ 2,276.3 Mn in terms of value by the end of 2017. The global spinal cord trauma treatment market is expected to expand at a CAGR of 3.7% over the forecast period to reach a value of US$ 3,036.2 Mn by 2025end.

Global Spinal Cord Trauma Treatment Market: Trends

Global Spinal Cord Trauma Treatment Market: Forecast by End User

On the basis of end user, the global spinal cord trauma treatment market is segmented into hospitals and trauma centers. Hospitals segment dominated the global spinal cord trauma treatment market in revenue terms in 2016 and is projected to continue to do so throughout the forecast period.

Hospitals and trauma centers segments are expected to approximately similar attractive index. Hospitals segment accounted for 53.2% value share in 2017 and is projected to account for 52.5% share by 2025 end.

Access Full Report @ https://www.persistencemarketresearch.com/checkout/17353

Global Spinal Cord Trauma Treatment Market: Forecast by Injury Type

On the basis of injury type, the global spinal cord trauma treatment market is segmented into complete spinal cord injuries and partial spinal cord injuries.

Partial spinal cord trauma treatment segment is expected to show better growth than the completed spinal cord treatment segment due to higher growth in the incidence rate of partial spinal cord trauma than the complete spinal cord trauma. With US$ 1,870.3 Mn market value in 2025, this segment is likely to expand at CAGR 3.8% throughout the projected period.

Global Spinal Cord Trauma Treatment Market: Forecast by Treatment Type

On the basis of treatment type, the global spinal cord trauma treatment market is segmented into corticosteroid, surgery, and spinal traction segments.

Surgery segment dominated the global spinal cord trauma treatment market in revenue terms in 2016 and is projected to continue to do so throughout the forecast period. Surgery segment is the most attractive segment, with attractiveness index of 2.6 over the forecast period.

Global Spinal Cord Trauma Treatment Market: Forecast by Region

This market is segmented into five regions such as North America, Latin America, Europe, APAC and MEA. Asia-Pacific account for the largest market share in the global spinal cord trauma treatment market.

Large patient population due to the high rate of road accidents and crime is making the Asia Pacific region most attractive market for spinal cord trauma treatment. On the other hand, MEA and Latin America is expected to be the least attractive market for spinal cord trauma treatment, with attractiveness index of 0.3 and 0.5 respectively over the forecast period.

Explore Extensive Coverage of PMR`s Life Sciences & Transformational Health Landscape

Molecular Diagnostics Market

Molecular Diagnostics Market Segmentation by Key Players Novartis AG, Roche Diagnostics, QIAGEN, Siemens Healthcare, Abbott Laboratories, Inc., Gen-Probe, Inc. (Hologic Inc.), Cepheid, Inc., Beckman Coulter, Inc., Becton, Dickinson & Company, Myriad Genetics, Inc., and bioMerieux.

Next-Generation Sequencing Market

Next Generation Sequencing Market Segmented by(By Application Whole-genome Sequencing ,Exome Sequencing,Targeted Resequencing,De Novo Sequencing,RNA Sequencing,ChIP Sequencing,Methyl Sequencing,Others);By Technology- Targeted Sequencing & Resequencing,Whole Genome Sequencing,Whole Exome Sequencing.

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Spinal Cord Trauma Treatment Market to Grow at Stellar CAGR 3.7% During the Forecast Period 2025 - MENAFN.COM

Spinal Cord Trauma Treatment Market: Global Industry Analysis 2012 2016 and Forecast 2017 2025is the recent report of Persistence Market Research that throws light on the overall market scenario during the period of eight years, i.e. 2017-2025. According to this report, Globalspinal cord trauma treatment marketis expected to witness significant growth during the forecast period.

This growth is expected to be primarily driven by increasing incidence of spinal cord trauma, and increasing government support to reduce the burden of spinal cord injuries. Additionally, development of nerve cells growth therapy is expected to boost the market in near future.

Get Sample Copy of Report @ https://www.persistencemarketresearch.com/samples/17353

Company Profiles

Get To Know Methodology of Report @ https://www.persistencemarketresearch.com/methodology/17353

The global market for spinal cord trauma treatment is is estimated to be valued at US$ 2,276.3 Mn in terms of value by the end of 2017. The global spinal cord trauma treatment market is expected to expand at a CAGR of 3.7% over the forecast period to reach a value of US$ 3,036.2 Mn by 2025end.

Global Spinal Cord Trauma Treatment Market: Trends

Global Spinal Cord Trauma Treatment Market: Forecast by End User

On the basis of end user, the global spinal cord trauma treatment market is segmented into hospitals and trauma centers. Hospitals segment dominated the global spinal cord trauma treatment market in revenue terms in 2016 and is projected to continue to do so throughout the forecast period.

Hospitals and trauma centers segments are expected to approximately similar attractive index. Hospitals segment accounted for 53.2% value share in 2017 and is projected to account for 52.5% share by 2025 end.

Access Full Report @ https://www.persistencemarketresearch.com/checkout/17353

Global Spinal Cord Trauma Treatment Market: Forecast by Injury Type

On the basis of injury type, the global spinal cord trauma treatment market is segmented into complete spinal cord injuries and partial spinal cord injuries.

Partial spinal cord trauma treatment segment is expected to show better growth than the completed spinal cord treatment segment due to higher growth in the incidence rate of partial spinal cord trauma than the complete spinal cord trauma. With US$ 1,870.3 Mn market value in 2025, this segment is likely to expand at CAGR 3.8% throughout the projected period.

Global Spinal Cord Trauma Treatment Market: Forecast by Treatment Type

On the basis of treatment type, the global spinal cord trauma treatment market is segmented into corticosteroid, surgery, and spinal traction segments.

Surgery segment dominated the global spinal cord trauma treatment market in revenue terms in 2016 and is projected to continue to do so throughout the forecast period. Surgery segment is the most attractive segment, with attractiveness index of 2.6 over the forecast period.

Global Spinal Cord Trauma Treatment Market: Forecast by Region

This market is segmented into five regions such as North America, Latin America, Europe, APAC and MEA. Asia-Pacific account for the largest market share in the global spinal cord trauma treatment market.

Large patient population due to the high rate of road accidents and crime is making the Asia Pacific region most attractive market for spinal cord trauma treatment. On the other hand, MEA and Latin America is expected to be the least attractive market for spinal cord trauma treatment, with attractiveness index of 0.3 and 0.5 respectively over the forecast period.

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Demand for Spinal Cord Trauma Treatment Market to Increase from End-use Industries During COVID-19 Pandemic and Substantially Surge Revenues in the...

-Spinal cord injury (SCI) creates a complex microenvironment that is not conducive to repair; growth factors are in short supply, whereas factors that inhibit regeneration are plentiful. In a new report, researchers have developed a structural bridge material that simultaneously stimulates IL-10 and NT-3 expression using a single bi-cistronic vector to alter the microenvironment and enhance repair. The article is reported in Tissue Engineering, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. Click here to read the article for free on the Tissue Engineering website through May 17, 2020.

In Polycistronic Delivery of IL-10 and NT-3 Promotes Oligodendrocyte Myelination and Functional Recovery in a Mouse Spinal Cord Injury Model, Lonnie D. Shea, PhD, University of Michigan, and coauthors report the development of a new poly(lactide-co-glycolide) (PLG) bridge with an incorporated polycistronic IL-3/NT-3 lentiviral construct. This material was used to stimulate repair in a mouse SCI model. IL-10 was included to successfully stimulate a regenerative phenotype in recruited macrophages, while NT-3 was used to promote axonal survival and elongation. The combined expression was successful; axonal density and myelination were increased, and locomotor functional recovery in mice was improved.

Inflammation plays a vital role in tissue repair and regeneration, and the use of a PLG bridge to take advantage of the inflammatory response to promote SCI repair is an elegant way to take advantage of these natural processes to improve SCI healing, says Tissue Engineering Co-Editor-in-Chief Antonios G. Mikos, PhD, Louis Calder Professor at Rice University, Houston, TX.

About the Journal

Tissue Engineering is an authoritative peer-reviewed journal published monthly online and in print in three parts: Part A, the flagship journal published 24 times per year; Part B: Reviews, published bimonthly, and Part C: Methods, published 12 times per year. Led by Co-Editors-in-Chief Antonios G. Mikos, PhD, Louis Calder Professor at Rice University, Houston, TX, and John P. Fisher, PhD, Fischell Family Distinguished Professor & Department Chair, and Director of the NIH Center for Engineering Complex Tissues at the University of Maryland, the Journal brings together scientific and medical experts in the fields of biomedical engineering, material science, molecular and cellular biology, and genetic engineering. Leadership of Tissue Engineering Parts B (Reviews) and Part C (Methods) is provided by Katja Schenke-Layland, PhD, Eberhard Karls University, Tbingen, Heungsoo Shin, PhD, Hanyang University; and John A. Jansen, DDS, PhD, Radboud University, and Xiumei Wang, PhD, Tsinghua University respectively. Tissue Engineering is the official journal of the Tissue Engineering & Regenerative Medicine International Society (TERMIS). Complete tables of content and a sample issue may be viewed on the Tissue Engineering website.

About the Publisher

Mary Ann Liebert, Inc., publishers is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Stem Cells and Development, Human Gene Therapy, and Advances in Wound Care. Its biotechnology trade magazine, GEN (Genetic Engineering & Biotechnology News), was the first in its field and is today the industrys most widely read publication worldwide. A complete list of the firms 90 journals, books, and newsmagazines is available on the e Mary Ann Liebert, Inc., publishers website.

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Co-delivery of IL-10 and NT-3 to Enhance Spinal Cord Injury Repair - Mirage News

We've heard that elderly people and those with underlying health conditions are most at risk if they're infected with coronavirus, but those can seem like really general terms. Who does that include? And why can they face more serious illness?"According to the , some of the underlying conditions that may put you at higher risk include: chronic lung disease and asthma, heart disease and undergoing cancer treatment," said CNN Chief Medical Correspondent Dr. Sanjay Gupta. Anyone with diabetes, kidney failure or liver failure may also be at higher risk.The role of the immune system is to protect against disease or other potentially damaging pathogens. A strong one is needed to help stave off coronavirus infection."Think of it like this," Dr. Gupta suggested. "In your everyday life, you're always fighting off pathogens. Most of the time you don't even realize it. If you have an underlying condition, it makes it more challenging to fight off a virus like this. You may develop a fever, shortness of breath or a cough more easily than someone who doesn't have a preexisting illness."Additionally, there are more specific reasons why each condition has its own vulnerabilities. Here's a guide to underlying conditions affected by coronavirus and why, and how you can protect yourself or an at-risk loved one.Older adultsEight out of 10 deaths reported in the U.S. have been in adults ages 65 and older, according to the CDC. Older adults have also been more likely to require hospitalization and admission to an intensive care unit.Older adults are more likely to have long-term health problems that can increase their risk for infection and serious disease. And, our immune systems usually weaken with age, making it more difficult for people to fight off infections, according to Johns Hopkins Medicine.The quality of our lung tissue also declines over time, becoming more elastic and making respiratory diseases such as COVID-19 of important concern because of the potential for lung damage.Inflammation in older adults can be more intense, leading to organ damage.Those with lung disease, asthma or heart conditionsPeople with chronic airway and lung diseases such as chronic obstructive pulmonary disease, asthma, pulmonary fibrosis and interstitial lung disease can lay the foundations for more severe infection with coronavirus because of the inflammation, scarring and lung damage those conditions cause, Johns Hopkins Medicine reported.COVID-19 affects a person's airway and lungs, but those organs work together to provide the body with oxygen. When the lungs are overburdened with an infection, the heart has to work harder, which exacerbates the challenges of people already living with heart disease.The immunocompromisedAccording to the CDC, many conditions can cause a person to be immunocompromised, including cancer treatment, smoking, bone marrow or organ transplantation and immune deficiencies. Poorly controlled HIV or AIDS and prolonged use of man-made steroid hormones or other immune-weakening medications can also hamper a person's immune function.Cancer can weaken immunity by spreading into the bone marrow, which makes blood cells that help fight infection, according to Cancer Research UK. Cancer prevents bone marrow from making enough blood cells.Some cancer treatments can temporarily weaken the immune system, too. Because cancer treatments such as chemotherapy, cancer drugs, radiotherapy or steroids are targeted toward cancer cells, they can also diminish the number of white blood cells created in the bone marrow.A 2017 study found cigarette smoking can harm the immune system by either causing extreme immune responses to pathogens or rendering the body less effective at fighting disease. This may occur by smoking, negatively altering the cellular and molecular mechanisms responsible for keeping an immune system strong.When a person undergoes a bone marrow transplant using stem cells from a donor, or they receive an organ, a doctor may prescribe medications to prevent graft-versus-host disease and mitigate the immune system's reaction by suppressing its function. After the operation, it takes time for your immune system to be up and running again.HIV and AIDS attack the body's immune system, specifically the body's T cells, which help the immune system fight off infection. When the diseases are untreated, HIV reduces the number of those cells, making the person more likely to contract other infections or infection-related cancer, according to the CDC.Severe obesityPeople with severe obesity, or a body mass index of 40 or higher, are at higher risk of serious disease."Obesity shares with most chronic diseases the presence of an inflammatory component," a 2012 study said. Inflammatory responses were linked between the immune system and body fat. Obesity is known to impair immune function by altering white blood cell count as well as the cells that control immune responses.DiabetesPeople with type 1 or type 2 diabetes face an increased risk of getting really sick with COVID-19, as both cause a blood sugar spike. If blood sugar is poorly managed, viral diseases can be more dangerous as high blood sugar may give viruses a place to thrive, according to Diabetes in Control, a news and information resource for medical professionals.Higher levels of inflammation have been discovered in the bodies of people with diabetes, weakening the immune system and making it more difficult for those affected to stave off sickness in general.Kidney and liver diseaseThe kidneys produce several hormones that affect immune responses. Having kidney disease and failure can weaken your immune system, making it easier for infections to take hold. According to the National Kidney Foundation, doctors and researchers have found that most infections are worse in people with kidney disease.The liver is an integral member of the body's line of defense, helping to regulate the number of white blood cells utilized in immune responses and defend against harmful pathogens. Someone with liver disease is experiencing abnormalities in the function of the immune system, giving rise to more serious illness.Neurodevelopmental conditionsNeurological and neurodevelopmental conditions may also increase the risk of serious COVID-19 for people of any age.These include disorders of the brain, spinal cord, peripheral nerve and muscle such as cerebral palsy, epilepsy, stroke and intellectual disability, according to the CDC. Those with moderate to severe developmental delay, muscular dystrophy or spinal cord injury are also more at-risk.People with neurological conditions may not be more at risk due to solely their condition, but because medications they might take to control their condition could hamper their immune system. However, some neurological conditions, such as Parkinson's, have been recognized to have inflammatory components, which may harm the immune system.Others including muscular dystrophy, multiple sclerosis or amyotrophic lateral sclerosis (ALS) could cause paralysis to the diaphragm, which leaves those affected very at risk for respiratory failure if they were to be sick with COVID-19.Staying safe when you're more at riskIf you see yourself on the list of those at higher risk for severe illness, there are several things you can do to protect yourself. First, make sure you are contact your doctor or doctors about your risk level. Second, be extra vigilant about the recommendations that most people are being asked to follow.Stay home whenever possible and avoid close contact with people, the CDC suggests. Wash your hands often to prevent transferring the virus from a surface to your face, and try to clean and disinfect frequently touched surfaces as often as you can.If you don't have an underlying condition, doing your part by practicing these cautionary measures can help protect not only you, but your loved ones with existing conditions.

We've heard that elderly people and those with underlying health conditions are most at risk if they're infected with coronavirus, but those can seem like really general terms. Who does that include? And why can they face more serious illness?

"According to the [Centers for Disease Control and Prevention], some of the underlying conditions that may put you at higher risk include: chronic lung disease and asthma, heart disease and undergoing cancer treatment," said CNN Chief Medical Correspondent Dr. Sanjay Gupta. Anyone with diabetes, kidney failure or liver failure may also be at higher risk.

The role of the immune system is to protect against disease or other potentially damaging pathogens. A strong one is needed to help stave off coronavirus infection.

"Think of it like this," Dr. Gupta suggested. "In your everyday life, you're always fighting off pathogens. Most of the time you don't even realize it. If you have an underlying condition, it makes it more challenging to fight off a virus like this. You may develop a fever, shortness of breath or a cough more easily than someone who doesn't have a preexisting illness."

Additionally, there are more specific reasons why each condition has its own vulnerabilities. Here's a guide to underlying conditions affected by coronavirus and why, and how you can protect yourself or an at-risk loved one.

Eight out of 10 deaths reported in the U.S. have been in adults ages 65 and older, according to the CDC. Older adults have also been more likely to require hospitalization and admission to an intensive care unit.

Older adults are more likely to have long-term health problems that can increase their risk for infection and serious disease. And, our immune systems usually weaken with age, making it more difficult for people to fight off infections, according to Johns Hopkins Medicine.

The quality of our lung tissue also declines over time, becoming more elastic and making respiratory diseases such as COVID-19 of important concern because of the potential for lung damage.

Inflammation in older adults can be more intense, leading to organ damage.

People with chronic airway and lung diseases such as chronic obstructive pulmonary disease, asthma, pulmonary fibrosis and interstitial lung disease can lay the foundations for more severe infection with coronavirus because of the inflammation, scarring and lung damage those conditions cause, Johns Hopkins Medicine reported.

COVID-19 affects a person's airway and lungs, but those organs work together to provide the body with oxygen. When the lungs are overburdened with an infection, the heart has to work harder, which exacerbates the challenges of people already living with heart disease.

According to the CDC, many conditions can cause a person to be immunocompromised, including cancer treatment, smoking, bone marrow or organ transplantation and immune deficiencies. Poorly controlled HIV or AIDS and prolonged use of man-made steroid hormones or other immune-weakening medications can also hamper a person's immune function.

Cancer can weaken immunity by spreading into the bone marrow, which makes blood cells that help fight infection, according to Cancer Research UK. Cancer prevents bone marrow from making enough blood cells.

Some cancer treatments can temporarily weaken the immune system, too. Because cancer treatments such as chemotherapy, cancer drugs, radiotherapy or steroids are targeted toward cancer cells, they can also diminish the number of white blood cells created in the bone marrow.

A 2017 study found cigarette smoking can harm the immune system by either causing extreme immune responses to pathogens or rendering the body less effective at fighting disease. This may occur by smoking, negatively altering the cellular and molecular mechanisms responsible for keeping an immune system strong.

When a person undergoes a bone marrow transplant using stem cells from a donor, or they receive an organ, a doctor may prescribe medications to prevent graft-versus-host disease and mitigate the immune system's reaction by suppressing its function. After the operation, it takes time for your immune system to be up and running again.

HIV and AIDS attack the body's immune system, specifically the body's T cells, which help the immune system fight off infection. When the diseases are untreated, HIV reduces the number of those cells, making the person more likely to contract other infections or infection-related cancer, according to the CDC.

People with severe obesity, or a body mass index of 40 or higher, are at higher risk of serious disease.

"Obesity shares with most chronic diseases the presence of an inflammatory component," a 2012 study said. Inflammatory responses were linked between the immune system and body fat. Obesity is known to impair immune function by altering white blood cell count as well as the cells that control immune responses.

People with type 1 or type 2 diabetes face an increased risk of getting really sick with COVID-19, as both cause a blood sugar spike. If blood sugar is poorly managed, viral diseases can be more dangerous as high blood sugar may give viruses a place to thrive, according to Diabetes in Control, a news and information resource for medical professionals.

Higher levels of inflammation have been discovered in the bodies of people with diabetes, weakening the immune system and making it more difficult for those affected to stave off sickness in general.

The kidneys produce several hormones that affect immune responses. Having kidney disease and failure can weaken your immune system, making it easier for infections to take hold. According to the National Kidney Foundation, doctors and researchers have found that most infections are worse in people with kidney disease.

The liver is an integral member of the body's line of defense, helping to regulate the number of white blood cells utilized in immune responses and defend against harmful pathogens. Someone with liver disease is experiencing abnormalities in the function of the immune system, giving rise to more serious illness.

Neurological and neurodevelopmental conditions may also increase the risk of serious COVID-19 for people of any age.

These include disorders of the brain, spinal cord, peripheral nerve and muscle such as cerebral palsy, epilepsy, stroke and intellectual disability, according to the CDC. Those with moderate to severe developmental delay, muscular dystrophy or spinal cord injury are also more at-risk.

People with neurological conditions may not be more at risk due to solely their condition, but because medications they might take to control their condition could hamper their immune system. However, some neurological conditions, such as Parkinson's, have been recognized to have inflammatory components, which may harm the immune system.

Others including muscular dystrophy, multiple sclerosis or amyotrophic lateral sclerosis (ALS) could cause paralysis to the diaphragm, which leaves those affected very at risk for respiratory failure if they were to be sick with COVID-19.

If you see yourself on the list of those at higher risk for severe illness, there are several things you can do to protect yourself. First, make sure you are contact your doctor or doctors about your risk level. Second, be extra vigilant about the recommendations that most people are being asked to follow.

Stay home whenever possible and avoid close contact with people, the CDC suggests. Wash your hands often to prevent transferring the virus from a surface to your face, and try to clean and disinfect frequently touched surfaces as often as you can.

If you don't have an underlying condition, doing your part by practicing these cautionary measures can help protect not only you, but your loved ones with existing conditions.

Read more:
What are the underlying conditions causing more serious illness from coronavirus? - WPBF West Palm Beach

Coronavirus (Image source: U.S. Department of State)

In my February column, I wrote about the fact that I had a stem cell transplant in early December 2019, about a month before I heard for the first time about the coronavirus.

The transplant entailed getting an unrelated donors stem cells to replace mine; then, if all went according to plan, these cells would grow into a new immune system to seek and destroy my cancer cells.

As a result of the transplant, all of my childhood vaccinations became ineffective. I was instructed to stay in isolation for at least four months in order to avoid infectious and possibly deadly diseases like influenza. Consequently, I have been quarantined since December.

Just a day before writing this, a friend told me that Im a trendsetter.

I knew very little about viruses before the coronavirus came alongonly that they were microscopic infectious organisms that invade living cells and then reproduce. In an effort to review what I had been (mostly unconsciously) protected from before transplant, I Googled the Centers for Disease Control and Prevention (CDC) and found a piece entitled, Vaccines for children: Diseases you almost forgot about.

I was reminded that most of us had vaccines as children for some of the nastiest viruses, including polio, which invades the brain and spinal cord and leads to paralysis; tetanus, a potentially fatal disease that causes lockjaw; whooping cough, which can lead to violent coughing that makes it difficult to breathe; and many more.

Most older adults are familiar with chicken pox, mumps and measles. I had two of them as a young teenager. One that I forgot about is diphtheria, which affects breathing or swallowing and can lead to heart failure, paralysis and death. There are several more.

I imagined the panic that parents must have felt and the pain that young children must have experienced before vaccines were discovered to prevent these horrible infectious diseases.

For the time being, I cannot replace my old vaccines. I must wait for at least one year while my new immune system gets stronger.

The idea of being in isolation and maintaining a safe social distance for a few months post-transplant made sense to me. I was well prepared by doctors and nurses and I knew my wife would be a great caregiver, so I thought I could do the time.

And then, the coronavirus came along.

For me, being quarantined was an old hat by the time a national emergency was declared and everything started to shut down. I learned that this new virus main target was the lungs and people older than 60 years with underlying health conditions were its primary targets.

I fit the bill and knew that Id have to do more time: at least another three months, my transplant doctor told me. The only difference is that this time, hundreds of millions of people would be joining me.

I was well-prepared before and after my transplant. I knew why I had to self-isolate and for how long. No one, including me, was prepared for COVID-19 and the mass quarantine that it now requiresnot only to protect oneself and ones family, but also to protect strangers. Mostly older strangers like me.

Scientists and other health professionals were the heroes of viral epidemics gone by. I do believe we will get through this, with people like immunologist Dr. Anthony Fauci leading the way.

Still, the unknown is what is most frightening. We all want answers, yet some remain illusive at the moment. This is an opportunity for all of us to strengthen our tolerance for ambiguity.

When will this end? No clue. Will it come back? No idea.

Although my new immune system needs more time to protect me, I just found out after a PET scan that Im in complete remission from my cancer.

Will it come back? No idea.

We are all in the same boat, living in uncertainty, whether young or old, healthy or unwell. As Plato said, Be kind, for everyone you meet is fighting a harder battle.

Andrew Malekoff is the executive director of North Shore Child and Family Guidance Center. To find out more, call 516-626-1971 or visit http://www.northshorechildguidance.org.

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A Quarantine Trendsetter - Long Island Weekly News