DUBLIN--(BUSINESS WIRE)--Horizon Therapeutics plc (Nasdaq: HZNP) today announced that it has submitted a regulatory filing to the Brazil National Health Surveillance Agency (ANVISA) for UPLIZNA for the treatment of adult patients with anti-aquaporin-4 immunoglobulin G seropositive (AQP4-IgG+) neuromyelitis optica spectrum disorder (NMOSD).

This regulatory submission is an important milestone as we continue to expand our commitment to NMOSD patients around the world, said Vikram Karnani, executive vice president and president, international operations, Horizon. NMOSD is a devastating disease with unpredictable attacks, which can result in potential loss of vision and motor function. We are hopeful that we can bring a potential new treatment option to the estimated ten thousand people living with NMOSD in Brazil.

In the N-MOmentum Phase 3 clinical trial, the largest NMOSD trial to date, UPLIZNA demonstrated a significant reduction in the risk of an NMOSD attack with only two infusions per year, following the initial two loading doses. Additionally, 89% of patients in the AQP4-IgG+ group remained attack-free during the six-month period post-treatment and 83% of patients on treatment remained attack-free for at least four years.1,2

UPLIZNA was approved by the U.S. Food and Drug Administration (FDA) in June 2020, by the Japanese Ministry of Health, Labor and Welfare in March 2021 and by the European Commission (EC) in April 2022. Mitsubishi Tanabe Pharma Corporation has the rights to develop and commercialize UPLIZNA in Japan, Thailand, South Korea, Indonesia, Vietnam, Malaysia, the Philippines, Singapore and Taiwan. Hansoh Pharmaceutical Group Company Limited, another strategic partner to Horizon, has also recently received manufacturing and marketing approval from the National Medical Products Administration of the Peoples Republic of China for UPLIZNA.

About Neuromyelitis Optica Spectrum Disorder (NMOSD)

NMOSD is a unifying term for neuromyelitis optica (NMO) and related syndromes. NMOSD is a rare, severe, relapsing, neuroinflammatory autoimmune disease that attacks the optic nerve, spinal cord, brain and brain stem.3-4 Approximately 80% of all patients with NMOSD test positive for anti-AQP4 antibodies.5 AQP4-IgG binds primarily to astrocytes in the central nervous system and triggers an escalating immune response that results in lesion formation and astrocyte death.6

Anti-AQP4 autoantibodies are produced by plasmablasts and plasma cells. These B-cell populations are central to NMOSD disease pathogenesis, and a large proportion of these cells express CD19.7 Depletion of these CD19 B cells is thought to remove an important contributor to inflammation, lesion formation and astrocyte damage. Clinically, this damage presents as an NMOSD attack, which can involve the optic nerve, spinal cord and brain.6-8 Loss of vision, paralysis, loss of sensation, bladder and bowel dysfunction, nerve pain and respiratory failure can all be manifestations of the disease.9 Each NMOSD attack can lead to further cumulative damage and disability.10,11 NMOSD occurs more commonly in women and may be more common in individuals of African and Asian descent.12,13

About UPLIZNA (inebilizumab-cdon)

INDICATION

UPLIZNA is indicated for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.

IMPORTANT SAFETY INFORMATION

UPLIZNA is contraindicated in patients with:

WARNINGS AND PRECAUTIONS

Infusion Reactions: UPLIZNA can cause infusion reactions, which can include headache, nausea, somnolence, dyspnea, fever, myalgia, rash or other symptoms. Infusion reactions were most common with the first infusion but were also observed during subsequent infusions. Administer pre-medication with a corticosteroid, an antihistamine and an anti-pyretic.

Infections: The most common infections reported by UPLIZNA-treated patients in the randomized and open-label periods included urinary tract infection (20%), nasopharyngitis (13%), upper respiratory tract infection (8%) and influenza (7%). Delay UPLIZNA administration in patients with an active infection until the infection is resolved.

Increased immunosuppressive effects are possible if combining UPLIZNA with another immunosuppressive therapy.

The risk of Hepatitis B Virus (HBV) reactivation has been observed with other B-cell-depleting antibodies. Perform HBV screening in all patients before initiation of treatment with UPLIZNA. Do not administer to patients with active hepatitis.

Although no confirmed cases of Progressive Multifocal Leukoencephalopathy (PML) were identified in UPLIZNA clinical trials, JC virus infection resulting in PML has been observed in patients treated with other B-cell-depleting antibodies and other therapies that affect immune competence. At the first sign or symptom suggestive of PML, withhold UPLIZNA and perform an appropriate diagnostic evaluation.

Patients should be evaluated for tuberculosis risk factors and tested for latent infection prior to initiating UPLIZNA.

Vaccination with live-attenuated or live vaccines is not recommended during treatment and after discontinuation, until B-cell repletion.

Reduction in Immunoglobulins: There may be a progressive and prolonged hypogammaglobulinemia or decline in the levels of total and individual immunoglobulins such as immunoglobulins G and M (IgG and IgM) with continued UPLIZNA treatment. Monitor the level of immunoglobulins at the beginning, during, and after discontinuation of treatment with UPLIZNA until B-cell repletion especially in patients with opportunistic or recurrent infections.

Fetal Risk: May cause fetal harm based on animal data. Advise females of reproductive potential of the potential risk to a fetus and to use an effective method of contraception during treatment and for 6 months after stopping UPLIZNA.

Adverse Reactions: The most common adverse reactions (at least 10% of patients treated with UPLIZNA and greater than placebo) were urinary tract infection and arthralgia.

For additional information on UPLIZNA, please see the Full Prescribing Information at http://www.UPLIZNA.com.

About Horizon

Horizon is a global biotechnology company focused on the discovery, development and commercialization of medicines that address critical needs for people impacted by rare, autoimmune and severe inflammatory diseases. Our pipeline is purposeful: We apply scientific expertise and courage to bring clinically meaningful therapies to patients. We believe science and compassion must work together to transform lives. For more information on how we go to incredible lengths to impact lives, visit http://www.horizontherapeutics.com and follow us on Twitter, LinkedIn, Instagram and Facebook.

Forward-Looking Statements

This press release contains forward-looking statements, including, but not limited to, statements related to potential regulatory approval of UPLIZNA in Brazil and the potential benefits of UPLIZNA to patients in Brazil. These forward-looking statements are based on management expectations and assumptions as of the date of this press release, and actual results may differ materially from those in these forward-looking statements as a result of various factors. These factors include the risk that UPLIZNA does not receive regulatory approval in Brazil, whether, if regulatory approval is received, UPLIZNA will be successfully commercialized in Brazil, and those risks detailed from time-to-time under the caption "Risk Factors" and elsewhere in Horizons filings and reports with the SEC. Horizon undertakes no duty or obligation to update any forward-looking statements contained in this press release as a result of new information.

References

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Horizon Therapeutics plc Submits Regulatory Filing for UPLIZNA (inebilizumab) in Brazil - Business Wire

Spinal Cord Stem Cells Comments Off on Horizon Therapeutics plc Submits Regulatory Filing for UPLIZNA (inebilizumab) in Brazil – Business Wire | June 20th, 2022

Abstract: Electrical stimulation influences neural stem cell neurogenesis. We analyzed the effects of electrical stimulation on neurogenesis in rodent spinal cord-derived neural stem cells (SC-NSCs) in vitro and in vivo and evaluated functional recovery and neural circuitry improvements with electrical stimulation using a rodent spinal cord injury (SCI) model. Rats (20 rats/group) were assigned to a sham (Group 1), SCI only (Group 2), SCI + electrode implant without stimulation (Group 3), and SCI + electrode with stimulation (Group 4) groups to count total SC-NSCs and differentiated neurons and evaluate morphological changes in differentiated neurons. Further, the Basso, Beattie, and Bresnahan scores were analyzed, and the motor and somatosensory evoked potentials in all rats were monitored. In vitro, biphasic electrical currents increased SC-NSC proliferation and neuronal differentiation and caused qualitative morphological changes in differentiated neurons. Electrical stimulation promoted SC-NSC proliferation and neuronal differentiation and improved functional outcomes and neural circuitry in SCI models. Increased Wnt3, Wnt7, and -catenin protein levels were also observed after electrical stimulation. In conclusion, our study proved the beneficial effects of electrical stimulation on SCI. We believe that Wnt/-catenin pathway activation may be associated with this relationship between electrical stimulation and neuronal regeneration after SCI.

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Effect of Electrical Stimulation on Spinal Cord Injury: In Vitro and In Vivo Analysis - Newswise

Spinal Cord Stem Cells Comments Off on Effect of Electrical Stimulation on Spinal Cord Injury: In Vitro and In Vivo Analysis – Newswise | June 11th, 2022

A 12-year-old boy's parents in the United Kingdom are trying to keep him hooked up to life support systems after doctors have said they believe he is "brain stem dead."

Archie Battersbee's mother and father, Holly Dance and Paul Battersbee want to give their son every chance at life after he was found unconscious on April 7 with a ligature around his neck. He reportedly had participated in what is believed to be an online blackout challenge, according to watchdog Christian Concern.

The boy is in critical condition at the Royal London Hospital.

His parents say a video of Archie gripping his mother's fingers is proof that he's still alive and his brain is functioning.

But his doctors believe there's no hope for the boy to recover since they believe his brain stem is dead. Scans show blood is not flowing to the area, according to Sky News. The stem lies at the base of the brain above the spinal cord. It is responsible for regulating most of the body's automatic functions essential for life. Doctors have said Archie's stem is 50% damaged and that 10% to 20% of the stem is in necrosis - where cells have died and/or are decaying.

Lawyers for the Barts Health NHS Trust said that doctors have repeatedly recreated the moment of the boy holding a clinician's hand, but the hospital workers felt "friction" not a grip, which the doctors say is consistent with muscle stiffness.

The hospital group has asked the Family Division of the High Court to rule that it is in Archie's 'best interests' to die by removing life support. However, Archie's family is not convinced that he is brain dead. They have experienced behavior that contradicts what the hospital first told them, and have also seen stories of remarkable recoveries from similar conditions in other patients, according to Christian Concern.

A High Court judge will decide if the boy will be taken off life support.

On Thursday, Archie's mother sat down for an interview with Christian Concern. She said she's fighting to keep her son alive.

Archie's mother Holly also told Sky News her son has not been given enough time to recover from his brain injury. "I don't understand the rush," she said. "I know they haven't got a lot of beds in hospital, but I don't understand the rush."

"I know he's in there and I know all that child needs is time. My gut instinct is spot on. My child is in there. He needs time to heal," she said.

An online petition to the hospital's chief executive officer has been created to ask that legal action be withdrawn in Archie's case. So far, almost 68,000 people have signed it.

Watch Christian Concern's video about Archie Battersbee below:

***Please sign up forCBN Newslettersand download theCBN News appto ensure you keep receiving the latest news from a distinctly Christian perspective.***

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UK Judge to Decide if 12-Year-Old Will Be Removed from Life Support, Parents Beg for More Time to Heal - CBN.com

Spinal Cord Stem Cells Comments Off on UK Judge to Decide if 12-Year-Old Will Be Removed from Life Support, Parents Beg for More Time to Heal – CBN.com | June 11th, 2022

Newswise LOS ANGELES (June 9, 2022) --Investigators from Cedars-Sinai will present the latest novel stem cell and regenerative medicine research at the International Society for Stem Cell Research (ISSCR) Annual Meeting, which is being held in person and virtually June 15-19 in San Francisco.

At this years scientific forum,Clive Svendsen, PhD, a renowned scientist and executive director of theCedars-SinaiBoard of Governors Regenerative Medicine Institute, willassume the role as treasurerfor the organization. In this position, he will be working with leading scientists, clinicians, business leaders, ethicists, and educators to pursue the common goal of advancing stem cell research and its translation to the clinic.

Along with taking on this leadership role, Svendsens work on a combination stem cell-gene therapy for the treatment of amyotrophic lateral sclerosis, afatal neurological disorder known as ALS or Lou Gehrig's disease, was selected as a Breakthrough Clinical Advances abstract and one ofthe years most compelling pieces of stem cell science. Svendsen will present data from the first spinal cord trial and a synopsis of the ongoing cortical trial and the potential impact this may have on this devastating disease.

The breakthrough oral session, A new trial transplanting neural progenitors modified to release GDNF into the motor cortex of patients with ALS, takes place on Thursday, June 16, from 5:15 to 7 p.m. The presentation is part of the Biotech, Pharma and AcademiaBringing Stem Cells to Patients Clinical Applications track.

Through this highly collaborative work, we hope to develop new therapeutic options for patients with such a debilitating and deadly disease, said Svendsen, who is also the Kerry and Simone Vickar Family Foundation Distinguished Chair in Regenerative Medicine.

All abstracts are embargoed until the start of each individual presentation.

Additional noteworthy presentations featuring Cedars-Sinai investigators at ISSCR 2022 include:

FollowCedars-Sinai Academic Medicineon Twitterfor more on the latest basic science and clinical research from Cedars-Sinai.

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First-of-its-Kind Stem Cell and Gene Therapy Highlighted at Annual Stem Cell Meeting - Newswise

Spinal Cord Stem Cells Comments Off on First-of-its-Kind Stem Cell and Gene Therapy Highlighted at Annual Stem Cell Meeting – Newswise | June 11th, 2022

Vertigo refers to a false sense of motion that can occur regardless of whether a person is moving. It is not a condition in itself but a possible symptom of several medical conditions.

Physical therapy may help a person reduce or eliminate vertigo. However, they should first speak with a doctor who can determine the underlying cause.

Once the doctor has confirmed a diagnosis, they may recommend physical therapy to help improve the persons symptoms.

This article explains how physical therapy can help people who experience vertigo. It also looks at exercises that a person can try at home and explains how to find a physical therapist.

Vertigo refers to a sensation of motion that is unrelated to the persons actions, and it typically presents as a spinning sensation. It may sometimes make a person feel as though their surroundings are spinning around them.

Vertigo is a symptom of other issues. However, it can also occur alongside or lead to other symptoms, such as balance issues, nausea, and motion sickness.

There are two types of vertigo: peripheral and central.

Peripheral vertigo accounts for about 80% of cases and is often the result of benign paroxysmal positional vertigo (BPPV).

The remaining 20% of cases are central vertigo, which results from lesions on the brain stem or another issue affecting the brain.

Both multiple sclerosis (MS) and migraine can cause central vertigo.

BPPV occurs when calcium carbonate crystals in the ear, known as canaliths, come loose and move into one of the fluid filled canals.

It is the most common cause of peripheral vertigo.

These crystals interfere with the normal movement of fluid in the canals. The purpose of the fluid is to sense movement, but disturbances can cause it to send false signals to the brain.

This tricks the brain into thinking that a person is moving, even if they are not. The false signal contradicts what the other ear senses and what the eyes are seeing. This conflicting information causes a spinning sensation, known as vertigo.

Physical therapy can help with vertigo. The most suitable exercises may vary depending on the type of vertigo. A person should make sure that they have the correct diagnosis before seeking physical therapy or trying exercises at home.

Healthcare professionals may use a form of physical therapy called vestibular rehabilitation therapy (VRT) to help with vertigo. VRT may help people with vertigo resulting from BPPV, head injuries, central nervous system lesions, and undefined causes.

However, this type of therapy might not work for all causes of vertigo. The aim of VRT is to help a person anticipate vertigo from known triggers and take action to prevent it from occurring. As a result, people who experience sporadic, unpredictable incidents may not benefit from VRT.

The symptoms of vertigo may either reduce or worsen during VRT exercises.

Sometimes, worsening symptoms may be due to unnecessary overuse of the exercises on a good day, which can cause fatigue, resulting in increased symptoms.

Even if the exercises seem to have resolved the symptoms of vertigo, a person can experience a relapse of symptoms at a later time.

Some exercises for vertigo may be easy for people to do at home. However, it is important to determine the cause of vertigo before beginning any therapy to treat the symptoms.

A person should also follow all exercise recommendations from a doctor or therapist. These professionals can explain each exercise in more detail and provide guidance on what to expect and when to stop.

This section explains how to perform two canalith repositioning exercises that may help alleviate vertigo.

Learn more about exercises for vertigo.

This common exercise is particularly effective in treating BPPV.

A person can perform the Epley maneuver by following these steps:

A person should then repeat the same movement on the opposite side in other words, facing the right at the beginning. They can do this up to three times per day until they no longer experience vertigo for at least 24 hours.

Learn more about the Epley maneuver with a step-by-step video guide.

This is a similar exercise that involves alternating between sitting and lying positions.

To perform Brandt-Daroff exercises, a person should:

Learn more about Brandt-Daroff exercises with a step-by-step video guide.

A person can ask a healthcare professional for their recommendations regarding physical therapists in the area. Not all therapists will have the same level of experience, and some may not know how to treat all causes of vertigo.

A person who needs help finding a physical therapist can use the Academy of Neurologic Physical Therapys website to find a local professional in their area.

The Vestibular Disorders Association also offers a resource that can help a person find physical therapists in their area.

The costs of physical therapy can vary, but health insurance may cover some or all of the costs. A person with a health insurance plan should contact their provider to determine how much of each session it will cover.

Those without insurance should talk with a healthcare professional, who may be able to provide information on local resources that can help cover the costs.

Learn more about Medicare and Medicaid.

Vertigo treatments can vary depending on the exact underlying cause. Once a person treats the underlying cause, the symptom of vertigo should resolve.

Other treatments that can help treat some causes of vertigo include:

Learn more about home remedies for vertigo.

With physical therapy and other effective treatments, most people should see their vertigo improve. A doctor can address any underlying conditions responsible for the vertigo.

However, a person may still experience some vertigo in the future. For example, about 50% of people will experience a relapse in BPPV within 5 years. In addition, about one-third of people experiencing vertigo from anxiety will still experience symptoms after 1 year.

Vertigo is a symptom associated with several different conditions. It occurs when a person experiences spinning and dizziness or feels as though their surroundings are moving around them.

Physical therapy can help improve a persons vertigo. A person should speak with a doctor before starting any new program to make sure that they receive effective treatment for the underlying condition.

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Physical therapy for vertigo: Exercises, benefits, and more - Medical News Today

Spinal Cord Stem Cells Comments Off on Physical therapy for vertigo: Exercises, benefits, and more – Medical News Today | May 29th, 2022

Danielle Roy says multiple sclerosis has turned her into a prisoner of her own body, which is why she is seeking a procedure that is only available outside of Canada and she needs the publics help to afford it.

The autoimmune disorder has slowly taken away her ability to walk and hold objects, leaving her wheelchair-bound after years of fighting to keep what mobility she has left. Roy said she is not giving up and is setting her sights on a stem-cell procedure that is still in the experimental phase in Canada but is being used in other countries to treat autoimmune disorders.

SUBMITTED

Danielle Roy is reaching out to the public to help her pay for an experimental stem-cell procedure in Mexico to halt the progression of her MS.

However, the nearest clinic is in Mexico, and it is going to cost her around $84,000. Neither Roy nor her caregiver and friend Evan Anthony have that kind of money, so they launched a campaign on GoFundMe to raise funds before applying for loans.

Roy said she knows asking for that much money is a lofty goal, but she has reached a point where she cannot tolerate her MS any longer.

"Im going to be bedridden soon. Im lucky I still have a lot of upper-body strength to get out of bed and into my chair," she said. "Really, I dont want to have to face another winter with this. For some reason, it makes my MS worse, and things really started going downhill after this winter."

What Roy is hoping to undergo is known as hematopoietic stem cell transplantation (HSCT). According to the medical information website Medscape.com, this involves injecting hematopoietic stem cells into the veins to re-establish blood-cell production in patients whose bone marrow or immune system is damaged or defective. This technique has been used with increasing frequency over the past 50 years to treat numerous malignant and non-malignant diseases.

Cells for HSCT may be obtained from the patient or from another person, such as a sibling or unrelated donor or an identical twin. Cell sources include bone marrow, peripheral blood and umbilical cord blood. Roy said the stem cells from her own body will be used.

According to the MS Society of Canada, the disease attacks the myelin, the protective covering of the brain and spinal cord, causing inflammation and often damaging the myelin in patches. When this happens, the usual flow of nerve impulses along nerve fibres (axons) is interrupted or distorted.

Depending on the type and the persons overall health, the result may be a wide variety of symptoms, depending on which part or parts of the central nervous system are affected. This includes numbness, loss of muscle control, paralysis, difficulty speaking, dizziness, loss of bowel and bladder control, difficulty swallowing and tremors. Not all people with MS will experience all symptoms, and often the symptoms will improve during periods of remission.

There are various ways to manage symptoms, ranging from drug treatments to non-medicinal strategies such as physiotherapy, occupational therapy, exercise programs and alternative and complementary treatments.

Roy was diagnosed in 2005 at the age of 19 and slowly lost mobility until she required an electric wheelchair. In 2010, she and her family ran a penny collection campaign to pay for a treatment anchored in the theory MS was caused by blocked neck veins that needed to be opened with angioplasty. At the time, such treatments were only available overseas.

Since then, it has been a series of ups and downs with several medications and therapies. The problem with those, she said, is they only slow down progression or manage symptoms for a time before they become worse.

The psychological effects have been just as devastating.

"I used to be so active, a cheerleader, a runner," she said. "Now, I feel a little jealous when I see someone holding a cup of coffee. This is my life, and Ill try anything to save it."

The hope is this treatment will stop the progression of MS and allow her body to heal itself and regain at least some of her mobility.

"Other treatments slow things down or do damage control, but with HSCT, it stops progression entirely," Anthony said. "Its not a treatment, but its hard to not call it a treatment. You can get it more than one time, but it is really meant to be a procedure done once."

Anthony said he can take out a loan to help pay for some of the procedure, but not for more than $84,000, which is why they are once again reaching out to the public to help Roy.

To donate, visit gofund.me/f3b0eaf8.

kmckinley@brandonsun.com

Twitter: @karenleighmcki1

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'This is my life, and I'll try anything to save it': Woman with MS raising funds for treatment - The Brandon Sun

Spinal Cord Stem Cells Comments Off on ‘This is my life, and I’ll try anything to save it’: Woman with MS raising funds for treatment – The Brandon Sun | May 29th, 2022

Neurosurgeons, researchers and engineers have come together to make it possible for a man who's a quadriplegic to drive a car using his brain.

On a track in Colorado Springs, racing thoughts and motor function have deeper meaning.

Quadriplegic German Aldana Zuniga lost movement after a car accident when he was 16 years old.

Now, he's the first patient to drive with his brain.

He can pull out of pit row, punch the throttle and speed away using only his mind and technology.

It all started with a question in 2013. Spinal cord neurosurgeon Dr. Scott Falci wondered, Could you modify a race car so people with spinal cord injuries can drive it?

"A large portion of this population that I was dealing with had a love affair for automobiles, cars, motor sports," Dr. Falci said. "I want to get spinal cord injured patients and just mobility-impaired patients in a race car where they can drive it themselves and just for the fun and the motivation and the inspiration that it would provide."

It could also serve as a real time lab. Engineers developed a modified driving system, using data from rides to improve the tech.

States away the Miami Project to end paralysis had their own research question: Could an FDA approved brain device for Parkinsons patients work for quadriplegics?

This is where Zuniga comes in. Miami Project doctors implanted that brain device in him and made a glove that connects with it.

Biomedical engineer Kevin Davis is part of the team.

Whenever hes thinking about moving his arm, we can detect a difference in the neural activity and that difference is what allows us to control external devices," Davis said.

The scientists joined forces with a new goal to combine both technologies.

"If we could harness the computational power of the brain, we could really take this quite far," Dr. Falci said.

Mind driving works like this: Zuniga forms a thought, like "open or look forward."

In the brain, that thought is a special signal with a unique electrical fingerprint. A part on the implanted device on top of his brain detects that signal and feeds it to computers in the car. Those computers are programmed to understand open and look forward and push the throttle and drive the car away.

Engineers swapped software code from Colorado to Florida while Zuniga drove a simulator for over a year. When it the time came for the real car:

"Its not even close, its totally different," Zuniga said. "You see the track, how big it is, the noise of the car, the heat of it."

"Over here when that is blue, hes thinking throttle off, when he goes green hes thinking throttle on, and youll see the numbers go up," Dr. Harry Direen said.

It's eight laps total, 850 horsepower, one quick water break.

Its the first time Zuniga has ever driven. He became a quadriplegic before he could get a drivers license.

"Once youre on the road, you feel the rush, the adrenaline," Zuniga said. "The track feels so short. I feel good, I feel fantastic, very happy."

The data from the track lab will go to improve the next ride, plus practical applications like controlling an electric wheelchair or an robotic prosthetic.

Dr. Falci is also researching how to restore spinal cord function with stem cells. It could bring back movement and feeling in the body.

"Regenerating the spinal cord, because that's the healthiest of all conditions," Dr. Falci said. "The more we can do for them or help them do on their own, the independence gained and the quality of life just goes up dramatically."

The road ahead for full restoration is a long one.

Dr. Falci has already spent 29 years working on it, but hes not gassed.

No matter how many more trips around the track it may take, theres one willing patient ready to propel forward.

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Racing Thoughts: Quadriplegic Man Drives Race Car With His Brain - Newsy

Spinal Cord Stem Cells Comments Off on Racing Thoughts: Quadriplegic Man Drives Race Car With His Brain – Newsy | May 29th, 2022

Astrocytes make up almost half of the mammalian brain cells. They are called glial cells because scientists originally thought that these starlight-shaped structures serve as nerve glue.

Research suggests that these cells control the growth of axons, or the neuronal projections that carry electrical impulses.

However, scientists still considered astrocytes to be supporting actors behind neurons, which are the primary cells of the brain and nervous system.

Now, scientists at Tufts University in Massachusetts and other institutions realize that astrocytes may execute a significantly greater performance in brain activity.

Dr. Moritz Armbruster, a research assistant professor of neuroscience at Tufts, led a team of researchers in harnessing novel technology to study astrocyte-neuron exchanges.

To their surprise, the scientists observed electrical activity in astrocyte processes within mouse brain tissue. They reported: This represents a novel class of subcellular astrocyte membrane dynamics and a new form of astrocyteneuron interaction.

Dr. Armbruster and his fellow authors published their findings in Nature Neuroscience.

Using innovative tools, the Tufts team developed a technique to detect and observe electrical activity in brain cell interactions. These properties could not be seen before now.

Dr. Chris Dulla, corresponding author of the study, is an associate professor of neuroscience at the Tufts University School of Medicine and Graduate School of Biomedical Sciences. He explained that he and his colleagues []use viruses to express fluorescent proteins in the mouse brain, and thats what lets us measure this activity.

In an interview with Medical News Today, he elaborated:

[W]e had other experiments that made us think that this new type of activity must be happening in astrocytes. We just didnt have a way to show it[] So, we developed these new techniques to image the activity of the astrocytes and, using them, we showed that this thing that we thought must be happening actually was happening.

Neurotransmitters are chemical messengers that facilitate the transfer of electrical signals between neurons and support the blood-brain barrier. Scientists have long understood that astrocytes control these substances to support neuronal health.

This study breaks ground in showing that neurons release potassium ions, which change the astrocytes electrical activity. This modulation affects how the astrocytes control neurotransmitters.

Until now, scientists could not image potassium activity in the brain.

Neurons and astrocytes talk with each other in a way that has not been known about before, Dr. Dulla said.

Dr. Dulla maintains that human brain cells work the same way as mouse tissue. He said that mouse and human brain cells use the same proteins and molecules involved in brain activity.

Besides, using human tissue samples presents ethical challenges, Dr. Dulla noted: [We] have to be really careful and judicious [] with the experiments we design, and [we] dont get a chance to see [human tissue] samples like [we] can do with mice.

However, the professor shared that extensive databases give [scientists] a chance to just access human brain tissue without doing an experiment [themselves], but just getting the data that someone else has already done.

This wealth of information further demonstrates similarities between human and mouse cells and lets researchers deduce that the same processes are happening in each. The main difference is that human cells are larger and more abundant.

He also pointed out that the study highlights a bidirectional relationship between these brain cells, as astrocytes influence the neurons as well.

These findings about astrocyte-neuron interactions open a new world of questions regarding brain pathology, memory, and learning.

MNT also discussed this study with Dr. Santosh Kesari, who was not involved in this research. He is a neurologist at Providence Saint Johns Health Center in Santa Monica, CA, and regional medical director for the Research Clinical Institute of Providence Southern California.

Dr. Kesari said that this study confirms earlier research.

[T]his is one of many studies thats showing increasingly, how astrocytes and neurons interact, how they affect each other and then connecting the dots to how that affects brain function behavior, memory, seizures, dementia, and even in the context of brain tumors, all these cells interact. Dr. Santosh Kesari

Most medication development for brain disorders currently targets neurons. Dr. Kesari agreed that this study might shine light on a new path.

Maybe we should really be understanding the astrocyte side of things to develop drugs that may impact brain health by looking at that astrocytic role in brain disorders, he said.

The ability to image cell processes, as in this study, makes it possible to explore other activities within the brain as well.

The researchers are also screening existing drugs in hopes of manipulating astrocyte-neuron processes. Scientists could come close to repairing brain injuries or helping people increase their learning capacity if this proves successful.

They are also making their tools available to other labs to explore more areas of interest, such as breathing, headache, and many other neurological disorders.

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Researchers find new function performed by almost half of brain cells - Medical News Today

Spinal Cord Stem Cells Comments Off on Researchers find new function performed by almost half of brain cells – Medical News Today | May 13th, 2022

A Texas family said they faced a fight for their childs life after their daughter's health plan provider denied coverage of a medication known as the most expensive drug in the world.

The family said doctors told them the one-time infusion that costs millions of dollars was their daughters best hope.

Read on to learn more about their story and how an "army" of strangers stepped in.

At 4-months old, every move Aniya Porter makes is a miracle to her parents.

Aniya Porter was born with a rare genetic disease called spinal muscular atrophy or SMA.

You go day-by-day. Is my daughter going to stop breathing? Is she going to stop attempting to stand up? Is she going to stop putting her head up? Will Porter said.

Aniya was born with a rare genetic disease called spinal muscular atrophy or SMA.

SMA progressively kills motor neurons, the nerve cells in the brain stem and spinal cord that control essential functions like talking, walking, swallowing and breathing.

We live every day wondering and hoping that nothing else happens to her, that she doesn't lose any more of her motor neurons, said Hailey Weihs, Aniyas mother.

There is treatment.

In 2019, the FDA approved a drug called Zolgensma which carries a price tag of $2.1 million. The one-time gene therapy, given through an IV, is designed to replace the function of a missing or non-working SMN1 gene in a patient to stop the progression of SMA preserving motor neurons before theyre gone.

It doesn't give back what she has already lost. That's why it's so time-sensitive, explained Weihs.

When Aniyas doctor prescribed Zolgensma, Weihs and Porter said their daughters insurance, a Medicaid provider in Texas, denied coverage of the drug.

She got the denial from her insurance and that was devastating for us, said Weihs.

As they appealed, they said Aniya started to show signs she may lose control of her muscles.

She started to stop having reflexes in her legs and then her tongue started twitching, said Porter.

Racing against time, Weihs connected with other families of kids with SMA and a lawyer who agreed to represent Aniya for free.

Aniyas attorney, Eamon Kelly, told NBC 5 Responds, All the doctors agree that Aniya Porter should receive this treatment.

Kelly, who is based in Chicago, said Aniya is the seventh child with SMA hes represented in insurance coverage battles.

In Aniyas case, he said the health plan provider said the treatment was not medically necessary because doctors believe Aniya has four copies of the SMN2 gene, also known as a backup gene, which can indicate a milder illness.

Kelly argued backup genes, along with other available therapies, would not be enough to keep Aniya from losing muscle function. However, he said cutting-edge gene therapy could help.

We have a treatment that will take a little girl that is going to have a degenerative disease that threatens her life, that will put her in a wheelchair and as long as we get it to her before she's two years old and before she loses her motor neurons, she'll walk, she'll dance, she'll live a full life. Its like science fiction, Kelly said.

The first child with SMA Kelly represented is Maisie Forrest, who received Zolgensma in 2019 when she was 20 months old.

Ciji Green, Maisies mom, said Maisie was on a ventilator 22 hours a day before getting the drug.

We met with her pulmonologist and I just wept, Green recalled. I told him she's not going to make it to two and he didn't offer any words of hope because he knew Maisie was on the decline as well.

Maisie is now 4 years old.

She touches my face and it's absolutely beautiful, said Green.

Maisie is playing, talking and crawling.

Something that I have now that I didn't have was hope, hope that I will get to see her continue to meet milestones that she was never supposed to meet, Green added.

Maisies mom and a team of volunteers known as Maisies Army introduced Aniyas parents to Eamon Kelly. Last month, Kelly represented Aniya at a Medicaid State Fair Hearing.

Aniyas family requested the hearing from Texas Health and Human Services.

A week after the hearing and a few days after NBC 5 reached out to Aniyas health plan provider, Superior HealthPlan, Superior told Aniyas family Zolgensma would be covered for Aniya.

We have fought for four months. Those have been the hardest four months of our lives, said Weihs.

On April 27, Aniyas family made the trip from their home in Abilene to Cook Childrens Medical Center in Fort Worth where Aniya received the drug.

She's going to sit up on her own, she is going to walk one day, she's going to feed herself with a spoon, she's going to walk down and she's going to get her diploma, Weihs said.

NBC 5 Responds reached out to Superior HealthPlan by phone and email. We didnt hear back.

We asked Texas Health and Human Services about options for families denied coverage for Zolgensma.

It told us, in part, Medicaid covers medically necessary services including medications, and those services are delivered through managed care organizations. If the prior authorization is denied, the provider or the member can appeal the decision and MCOs have flexibility to make medically necessary decisions. Members also have the right to access the State Fair Hearing process with or without an External Medical Review (EMR). The EMR is conducted by a third-party Independent Review Organization.

It also shared, Medicaid covers alternative therapies for spinal muscular atrophy treatment, including Spinraza (nusinersen) and Evrysdi (risdiplam). Also, some manufacturers offer patient assistance programs.

A spokesperson for Novartis, which makes Zolgensma, told NBC 5, in part, Zolgensma (onasemnogene abeparvovec) is a transformative and highly innovative gene therapy for a devastating, progressive genetic disease. This one-time gene therapy is priced based on the value it provides to patients, caregivers and health systems.

It also said, Novartis is working in partnership with governments and health care systems worldwide to identify and define new sustainable access models.

Aniyas parents said she will still see a doctor and be monitored after getting the gene therapy treatment. Theyre hopeful about her prognosis.

We just knew we couldnt give up, Weihs said.

Weihs tells NBC 5 Responds the family is now focused on helping other kids get access to a drug they believe is priceless.

I don't care how expensive it is. It's a child's life. Every baby deserves a chance, said Porter.

Novartis said more than 1,800 patients have been treated with Zolgensma worldwide.

Texas newborn screening program began screening for SMA last June. The states health and human services website reports SMA is among the leading genetic causes of death among infants and toddlers.

NBC 5 Responds is committed to researching your concerns and recovering your money. Our goal is to get you answers and, if possible, solutions and a resolution. Call us at 844-5RESPND (844-573-7763) orfill out our customer complaint form.

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Texas Family Fights to Access $2.1 Million Treatment for Baby - NBC 5 Dallas-Fort Worth

Spinal Cord Stem Cells Comments Off on Texas Family Fights to Access $2.1 Million Treatment for Baby – NBC 5 Dallas-Fort Worth | May 13th, 2022

A recent study appearing in the journaleClinical Medicinesuggests that severe COVID-19 may be associated with persistent cognitive deficits, equivalent to a decline of 10 IQ points. In this study, severe COVID-19 was defined as COVID-19 that required hospitalization and critical care.

These cognitive deficits persisted until at least 6 months after contracting the SARS-CoV-2 infection, with a gradual improvement, if any, in these cognitive symptoms. These results underscore the importance of longer-term support for patients who have recovered from severe COVID-19.

According to official data from 2020, which is the same year that this study drew its data from, about 4 in 10 adults over the age of 18 are at risk of developing severe COVID-19 in the United States.

A significant minority of individuals with a SARS-CoV-2 infection experience persistent cognitive symptoms following the initial 4 weeks after the onset of COVID-19 symptoms. Some of the common cognitive symptoms include problems with concentration, brain fog, memory, and executive function.

Although persistent cognitive symptoms are also observed in individuals with mild COVID-19, such deficits in cognitive function are more prevalent in individuals with severe COVID-19. Previous studies suggest that 36%76% of individuals with severe acute COVID-19 show cognitive deficits 6 months after illness onset.

However, further research is needed to understand the specific aspects of cognitive function that are affected after severe COVID-19 and the factors that predict these cognitive symptoms.

Previous studies characterizing persistent cognitive symptoms in COVID-19 patients have relied on self-reports, which are susceptible to bias. Other studies have used pen-and-paper neuropsychological tests to assess cognitive function.

However, these tests do not possess the sensitivity to detect small changes in cognitive function or distinguish the various domains or aspects of cognitive function impacted by a SARS-CoV-2 infection.

To address these concerns, the authors of the present study used computerized cognitive tests to objectively characterize specific domains of cognitive function impacted after severe acute COVID-19. These computerized tests also allowed the researchers to assess the magnitude of these cognitive deficits.

Individuals with COVID-19 also experience persistent mental health symptoms such as anxiety, depression, fatigue, and post-traumatic stress disorder (PTSD), which could contribute to the deficits in cognitive function.

Another objective of the present study was to determine whether these mental health symptoms mediate the persistent cognitive deficits in COVID-19 patients.

The present study involved 46 patients who were previously hospitalized for severe COVID-19 and received critical care in Addenbrookes Hospital in Cambridge, England. The former COVID-19 patients completed a series of computerized cognitive tests during a return visit to the hospital, an average of 6 months after the onset of the illness.

The performance of the 46 participants on the cognitive tests was compared with that of 460 individuals in the control group. The individuals in the control group were not hospitalized for COVID-19 and were matched for age, sex, and education levels. The researchers also used self-reports to assess symptoms of anxiety, depression, and PTSD.

The researchers found that the COVID-19 patients had a lower score and a slower response time in the cognitive tests than the matched controls. People who had COVID-19 showed more pronounced deficits in specific domains of cognition, including processing speed, attention, memory, reasoning, and planning.

Notably, the deficits in cognitive function in the COVID-19 survivors were not associated with mental health symptoms present at the time of the cognitive testing, such as depression, anxiety, and PTSD.

Instead, the performance in the cognitive tests was correlated with the severity of acute illness. For instance, cognitive deficits were more pronounced in individuals who required mechanical ventilation.

The researchers then compared the performance of COVID-19 survivors with over 66,000 individuals from the general population.

The magnitude of cognitive impairment in COVID-19 survivors was equivalent to the age-related cognitive decline expected during the 20year period between the ages of 50 and 70 years.

The studys lead author Professor David Menon, head of the Division of Anaesthesia at the University of Cambridge, says: Cognitive impairment is common to a wide range of neurological disorders, including dementia, and even routine aging, but the patterns we saw the cognitive fingerprint of COVID-19 was distinct from all of these.

Dr. Betty Raman, a cardiologist at the University of Oxford, told Medical News Today, This prospective cohort study of 46 individuals recovering from severe COVID-19 and large normative reference population by Hampshire and colleagues has shown a clear association between severity of infection and degree of cognitive impairment.

This multidimensional characterization of cognition provides a nuanced understanding of distinct patterns of cognitive impairment during the convalescent phase of severe COVID-19. Future efforts are needed to understand how this pattern varies in the context of other post-infectious syndromes and critical illness.

The study found that these cognitive deficits persisted until 6-10 months after the onset of COVID-19, and there was only a gradual improvement, if any, in cognitive performance. The persistence of these cognitive deficits highlights the importance of understanding the mechanisms underlying these symptoms.

Scientists have proposed multiple mechanisms, such as direct infection of the brain by SARS-CoV-2 and disruption of blood supply to the brain, to explain the persistent cognitive symptoms in COVID-19 patients. Among these mechanisms, systemic or whole-body inflammation has emerged as the leading candidate responsible for persistent cognitive symptoms.

Dr. Roger McIntyre, a professor of Psychiatry and Pharmacology at the University of Toronto, told MNT, Inflammatory activation appears to be mediating these findings, highlighting the hazards of lengthy immune activation. The next steps are to unravel biological mechanisms more fully and identify prevention and treatment strategies.

Discussing major questions that need to be addressed, Dr. Paul Harrison, a professor of psychiatry at the University of Oxford, said:

This study shows that these deficits can be substantial and persist more than 6 months after the acute illness. The results are convincing and important and raise further questions. For example, what happens following a less severe infection? How long do the deficits last? What causes them and, critically, how can they be treated or prevented?

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Severe COVID-19 may cause cognitive deficits equivalent to 20 years of aging - Medical News Today

Spinal Cord Stem Cells Comments Off on Severe COVID-19 may cause cognitive deficits equivalent to 20 years of aging – Medical News Today | May 13th, 2022

Stem cells are key building blocks for the human body. At the start of life, they divide over and over again to create a fully developed baby from an embryo. Many individuals now even turn to services that store and preserve umbilical cords should a person ever be in need.

Stem cells have the potential to develop into different types of cells in the body, serving as a repair system of sorts for damaged or lost cells. In recent decades, scientists have shown the miraculous ways of medicine through stem cell treatments.

So just how are doctors using stem cells to treat and help heal people battling various ailments? Heres a look at five studies published on StudyFinds that demonstrate the wondrous ways of stem cell treatments.

A heart condition called dilated cardiomyopathy, or DCM, weakens muscles of the ventricles, which causes heart failure and often death in children. Currently, the only cure is a heart transplant, which can take long periods of time to find an acceptable donor and increases the risk of rejection of the donor tissue. One study finds that stem cell therapy could help DCM patients survive longer while awaiting a transplant or potentially eliminate the need for a new heart entirely.

Cardiac stem cells called cardiosphere-derived cells (CDCs) have proven to be effective at treating certain heart conditions. The CDCs grow into tissue cells of the heart and can counter the effects of DCM. To test the safety of the CDC therapy, a team of scientists at Okayama University in Japan demonstrated the efficacy of CDCs in tissue damaged from DCM. For the study, DCM symptoms were induced in pigs, after which CDCs were administered in various doses for treatment. In a control group, some pigs were given a placebo.

Results showed thickening of the heart muscle in pigs who were given the stem cell treatment. This allows increased blood flowto the rest of the body, thereby effectively repairing the damaged tissue. Due to the dosage used in animal trials, researchers could estimate the proper dosage for human trials.

The first of these included 5 younger patients who were diagnosed with DCM. Injections of CDCs resulted inbetter heart function without any serious side effects. Thus, scientists believe this type of treatment could minimize the need for heart transplants and allow DCM patients to have normal lives.

READ MORE: Stem cell treatment shows promise as treatment for rare heart condition in children

Although their use is sometimes controversial, scientists often look at stem cells as a potential miracle cure for many conditions. One study finds stem cells from a babys umbilical cord may save the most at risk of dying from COVID-19. A treatment derived from non-altered versions of these stem cells significantly improves the survival rate among coronavirus patients already on a ventilator.

In a double-blind, controlled, randomized study, 40 adultpatients in intensive careand on a ventilator received the treatment intravenously. The infusions contained stem cells coming from the connective tissue of a human umbilical cord. Half of the patients received infusions not containing stem cells to serve as a control group.

Results reveal survival rates climbed by 2.5 times among patients receiving stem cells. Those with a pre-existing health problem, making them high-risk for COVID, saw their changes of beating coronavirus jump by 4.5 times. Moreover, the study says the stem cell infusions did not cause any life-threatening complications or allergic reactions.

READ MORE: Stem cells from a babys umbilical cord doubles survival chances among COVID patients

In the fight against heart disease, a new super-weapon is now even closer to deployment, and its capabilities are turning out to be beyond expectations. A study aimed at combating heart disease finds that stem cells are not only showing promise in treating heart failure, but in rats are actually reversing problems associated with old age.

The specific type of stem cells used in the study are cardiosphere-derived cells, or CDCs. While the latest research involving CDCs indicates possibilities that have previously been in the realm of science fiction, the scientists leading the charge urge restraint in face of the excitement.

Nevertheless, the latest results of stem cell infusions in rats are startling. Not only did rats that received the CDCs experience improved heart function, they also had lengthened heart cell telomeres. Moreover, the rats that received the treatment also had their exercise capacity increase by about 20 percent. They also regrew hair faster than rats that didnt receive the cells.

Still, the doctors and scientists working to push the frontier of medicine forward are very optimistic about the real possibilities of the therapy. Researchers of the study said they are also studying the use of stem cells in treating patients with Duchenne muscular dystrophy and patients with heart failure with preserved ejection fraction, a condition that affects more than 50 percent of all heart failure patients.

READ MORE: Study: Cardiac stem cell injections reverse effects of aging

A new biomaterial can help regenerate tissue in people dealing with chronic lower back pain and spinal issues. A recent study finds the secret to this breakthrough therapy is all in the hiPS. Not thosehips, but human induced pluripotent stem cells.

The study explains that a common cause of lower back pain is the degeneration of intervertebral discs (IVDs). These discs sit between the vertebrae in the spine and help give the spinal column its flexibility. Severe IVD degeneration eventually leads to spinal deformity without treatment. In this study, scientists used cartilage tissue derived from stem cells to build back lost IVDs in lab rats.

Study authors used induced pluripotent stem cells (iPSCs) during their experiments. Importantly, scientists are capable of turning iPSCs into chondrocytes cells that produce and maintain cartilage. Previous studies have successfully used this same method to treat cartilage defects in animals. In the new study, researchers created human iPSC-derived cartilaginous tissue (hiPS-Cart) that they implanted into rats with no NP cells in their intervertebral discs.

Findings reveal that the hiPS-Cart implanted in the rats was able to survive and be maintained. IVD and vertebral bone degeneration were prevented. The researchers also assessed the mechanics and found that hiPS-Cart was able to revert these properties to similar levels observed in the control rats.

READ MORE: Stem cell cure for lower back pain is all in the hiPS

Stem cells taken from deceased patients may also help in creating a cure for blindness. Retina cells from a corpse continue to survive after being transplanted into the eyes of monkeys, scientists say.

RPE dysfunction is a leading cause of blindness, including causing disorders likemacular degeneration, which affects around 200 million people worldwide. Now, for the first time, scientists have successfully produced retina cells in monkeys using human stem cells. Human cadaver donor-derived cells can be safely transplanted underneath the retina and replace host function, and therefore may be a promising source for rescuing visionin patients with retina diseases.

For the study, researchers transplanted stem cells from the eyes of donated bodies under the monkeys macula, the central part of the retina. Following surgery, the transplanted patches remained stable for at least three months without any serious side-effects. The RPE created by the human stem cells partially took over from the old retina cells. In addition, this could successfully support the eyes light receptorswithout causing retinal scarring.

These unique cells could serve as an unlimited resource of human RPE, whichmay restore sightfor millions of people around the world. The scientists caution that they will need to conduct more research to see how the procedure works with human transplant patients. Human trials are still a long way off.

READ MORE: Eye stem cells transplanted from corpses to live patients could cure blindness

For more information on each of these stem cell treatments, you can refer to the READ MORE links in between each section.

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Stem Cell Magic: 5 Promising Treatments For Major Medical Conditions - Study Finds

Spinal Cord Stem Cells Comments Off on Stem Cell Magic: 5 Promising Treatments For Major Medical Conditions – Study Finds | April 29th, 2022

When a freak accident left mum-of-three Sam Bloom paralysed from the waist down, she sunk into a deep depression before finding comfort from an unlikely source.

Bloom stumbled across a baby magpie she called Penguin Bloom, and the bird soon became a member of her family - eventually helping to assist in her recovery.

Her book of the same name later became a movie, starring Australian actress Naomi Watts as Sam.

Watch The Morning Show on Channel 7 and stream it for free on 7plus >>

See Sam Bloom in the video player above

But that is far from the end of Blooms remarkable story.

She is both a world para-surf champion and disability advocate, fighting for a cure for spinal cord injuries.

When she joined The Morning Show, she started by discussing the impact of the film.

I think the best thing about the whole film and telling our story is just all the messages Ive received from people around the world, Bloom said.

It was on Netflix in South America and Europe and so on, and a lot of people have just said thank you because they dont feel so alone because it was a pretty honest account of when life doesnt turn out the way you thought it would.

Its nice to know that youre actually helping someone.

Bloom was an avid surfer before her accident, and her injury hasnt deterred her. She has twice taken out the world para-surf championship and recently returned from a surfing trip with her family in Yeppoon in north Queensland.

It was so much fun, the wave pool at Yeppoon is like a giant lake and its like it has this giant plunger in the middle and it sucks up, Bloom said.

Its a bit terrifying at first and then there are five different breaks - it is the best fun ever.

I feel super-free, kind of feel like my old self again, which is a nice feeling. Its the best.

Bloom also spoke about her work with Wings for Life World Run - the worlds largest running event in which thousands of people around the globe run simultaneously to raise money and awareness for spinal cord injury research.

The run is on May 8 at 9am in Sydney and the run starts all around the world at the same time, in a way were lucky that its not in the middle of the night in Sydney when it starts, Bloom said with a laugh.

Bloom explained the goal behind the run and her hopes for what it might ultimately achieve: funding vital research into spinal injuries.

Unfortunately its all about money, she said.

Thats the best thing about Wings for Life World Run, 100 per cent of the money raised goes straight to the research.

Its incredible, theyre doing a lot of research now with stem cells and neurostimulation.

I hope that theyll find a cure for spinal cord injuries. Can you imagine, theres millions of people around the world living with spinal cord injury and it breaks my heart when I see young people (affected).

Because I was 41 when I had my accident and I know how devastating it is, when you see young people and their journey is only just beginning.

Bloom revealed that, while her outlook is a lot more positive, she still struggles with living with her injury

I have good days and bad days, for sure, she said. To be honest, I hate being stuck in a wheelchair, Id do anything to be up and to be me again.

To help support the Wings for Life World Run please click here

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Still Blooming: Sams mission to raise money for spinal cord injury research - 7NEWS

Spinal Cord Stem Cells Comments Off on Still Blooming: Sams mission to raise money for spinal cord injury research – 7NEWS | April 15th, 2022

Scientists have created a stem cell treatment that might potentially lead to novel restorative therapies for those who have suffered a spinal cord damage.

Clinical trials have been hindered by limited stem cell viability and inability to replace injured spinal cord cells following spinal cord damage, despite its enormous promise for tissue healing.

Using a tailored method, this study establishes ground-breaking new territory by directing grafted neuronal stem cells to produce the specific kinds of spinal cord repair cells. It is critical that these newly generated cells survive and operate inside the host wounded spinal cord for a lengthy period of time following a spinal cord accident.

As a neurodegenerative ailment, spinal cord damage is a severe and expensive one, Karimi noted. She estimates that roughly 1,400 new occurrences of spinal cord injury occur annually in Canada, out of a total population of 86,000 people. Of them, 40% are all below the age of 45. It is anticipated that in 2019, the yearly cost of spine nerve lesion in Canada would be around $2.7 billion. She said that these expenses include medical treatment and hospitalizations, and also indirect expenses such as missed or decreased output.

Developing innovative restorative medicine therapies to enhance the standard of life for a wide group of people is an unfulfilled need in the field of spinal cord injury rehabilitation. This is exciting news for spinal cord injury sufferers, who have seen few advancements in treatment since the advent of stem cell research.

It will likely still be some time before this kind of treatment is available to patients, but we know that the researchers involved in this study are doing everything they can to advance neural stem cell transplantation therapies and bring them to a wider clinical application.

The study was published in The Journal of Neuroscience.

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Neural Stem Cell Therapy For Spinal Cord Injury To Tap Into The Potential Of Stem Cells - Optic Flux

Spinal Cord Stem Cells Comments Off on Neural Stem Cell Therapy For Spinal Cord Injury To Tap Into The Potential Of Stem Cells – Optic Flux | April 15th, 2022

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Lineage and Cancer Research UK Announce Completion of Patient Enrollment in Phase 1 Clinical Study of VAC2 for the Treatment of Non-small Cell Lung...

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Lineage Announces Pipeline Expansion to Include Auditory Neuronal Cell Therapy for Treatment of Hearing Loss - Galveston County Daily News

Spinal Cord Stem Cells Comments Off on Lineage Announces Pipeline Expansion to Include Auditory Neuronal Cell Therapy for Treatment of Hearing Loss – Galveston County Daily News | March 22nd, 2022

All data and statistics are based on publicly available data at the time of publication. Some information may be out of date. Visit our coronavirus hub and follow our live updates page for the most recent information on the COVID-19 pandemic.

A recent study in Nature found subtle changes in the brains of people with mild to moderate COVID-19 after the initial 4 weeks or acute phase of a SARS-CoV-2 infection. The study showed that individuals with SARS-CoV-2 showed greater brain tissue damage and shrinkage of brain regions at an average of 4.5 months after their COVID-19 diagnosis.

Dr. Maxime Taquet, a senior research fellow at the University of Oxford, who was not involved in the study, said: It is well established that [SARS-CoV-2] infection is associated with subsequent risks of neurological and psychiatric problems in some people, including brain fog, loss of taste and smell, depression, and psychosis. But why this occurs remains largely unknown.

This study starts to shed light on this important question by showing that brain regions connected to the smell center of the brain can shrink after COVID-19 in some people.

The studys co-author, Professor Naomi Allen, chief scientist at UK Biobank, noted, [This] is the only study in the world to be able to demonstrate before versus after changes in the brain associated with SARS-CoV-2 infection.

Neurological symptoms are common both during and after the acute phase of a SARS-CoV-2 infection. Previous studies examining changes in the brain underlying these neurological symptoms have mostly focused on people with acute COVID-19.

The small number of studies assessing brain changes after the acute phase of a SARS-CoV-2 infection lacked access to brain imaging data before the infection. Consequently, some of the differences observable in these studies could be due to brain anomalies or risk factors that existed before the infection.

Researchers conducted the present study to distinguish brain anomalies relating to COVID-19 from those that may occur due to preexisting risk factors. Moreover, the study used multiple types of brain scans to assess brain changes in many individuals, facilitating the identification of subtle brain anomalies associated with the SARS-CoV-2 infection.

In the present study, the researchers used data from the UK Biobank, a large database containing medical information, including brain imaging data, from individuals in the United Kingdom.

Specifically, they used imaging data collected from 785 people using different brain scans before and after the onset of the COVID-19 pandemic. This included 401 participants with a SARS-CoV-2 infection between the two scans and 384 control adults without a COVID-19 diagnosis.

The scientists matched participants in the two groups for age, sex, ethnicity, and the duration between the two brain scans. The average duration between the COVID-19 diagnosis and the second set of brain scans was 141 days.

The researchers used software programs to analyze the raw brain imaging data and extract quantifiable features, called image-derived phenotypes (IDPs). Each IDP measures a specific brain structure or function, such as the change in brain region activity while performing a task or the volume of a specific brain structure.

In the present study, the researchers measured changes in over 2,500 IDPs for each individual.

A loss of smell or olfaction is observable in most individuals with a SARS-CoV-2 infection, including after the acute phase. Therefore, the researchers focused on brain regions either directly involved in processing olfactory information or those connected to the olfactory system.

They found a greater reduction in gray matter volume and a greater increase in tissue damage markers in specific brain regions associated with the olfactory system in participants with SARS-CoV-2 compared with controls. The gray matter comprises mainly of cell bodies of nerve cells and is involved in information processing.

There was also a greater loss of gray matter across the entire brain and an increase in the volume of cerebrospinal fluid in participants with a SARS-CoV-2 infection.

In other words, besides changes in brain regions associated with olfaction, there were global changes in the brains of participants with SARS-CoV-2. Notably, these brain anomalies were observable in individuals with mild to moderate COVID-19.

Examining differences in cognitive function, the researchers found that the participants with SARS-CoV-2 showed deficits in executive function, which encompasses higher-level cognitive functions such as thinking, reasoning, and decision-making.

Additionally, there was a correlation between a lower performance in the executive function test and atypical brain changes in a part of the cerebellum known to be involved in cognition.

These findings might help explain why some people experience brain symptoms long after the acute infection. The causes of these brain changes, whether they can be prevented or even reverted, as well as whether similar changes are observed in hospitalized patients, in children and younger adults, and in minority ethnic groups, remain to be determined, said Dr. Taquet.

However, the researchers noted that they did not have data on whether the participants with a SARS-CoV-2 infection had symptoms of long COVID. They were also unable to assess the association between the brain anomalies and potential long COVID symptoms.

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COVID-19: Even mild to moderate infection may cause brain anomalies - Medical News Today

Spinal Cord Stem Cells Comments Off on COVID-19: Even mild to moderate infection may cause brain anomalies – Medical News Today | March 22nd, 2022

A mum who has lived with multiple sclerosis for over a decade says a 50,000 treatment unavailable on the NHS could be her last shot at living a life largely unhindered by the disease.

Jodie McQuillian, 32, was formally diagnosed with the chronic condition in 2015, a few years after the first signs appeared when she temporarily lost vision in her left eye.

Since then, she has undergone multiple treatments and bouts of physiotherapy in order to stave off relapses of the condition.

But the mum of one faces life in a wheelchair if she can't put a halt to the rampant flare-ups.

Multiple sclerosis, often known as MS, is a condition where the immune system mistakenly attacks nerves around the brain and spinal cord, affecting the body's ability to transmit signals properly.

Each time Jodie "relapses" - when her body launches a new attack on itself - she finds herself sapped of energy and often experiences issues with her sight and mobility.

It takes her months to recover from each flare-up, affecting the time she can spend with son Ethan, five, and her family.

And every time there's a relapse, a little bit of her doesn't come back.

Jodie, from Alloa, told the Record: "I've just had another relapse and everything I'm trying isn't really effective enough.

"Since I started my newest treatment my walking has gotten a lot worse.

"Every time you relapse you recover but it takes months and you get put on a high dose of steroids and that drains you of all your muscle.

"I'm always left a wee bit damaged from a relapse - and when I feel like I've sort of recovered they flare up again."

After experiencing a major flare-up when she gave birth to Ethan, Jodie began undergoing treatment for MS, trying every drug available on the NHS in a bid to reduce the risk of relapsing.

Despite trying Copaxone injections, Tecfidera tablets and Ocrevus infusions through a drip in her arm over the course of five years, the setbacks have continued and Jodie's outlook is bleak.

There is currently no cure for MS and while her condition is currently recurring intermittently, it is likely to become progressive later in life with little hope of recovery.

However, her last hope may lie in a new treatment known as haematopoietic stem cell transplantation, or HSCT.

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Backed by the MS Society, it is an intense chemotherapy procedure that aims to "reset" Jodie's immune system and stop it from attacking her brain and spinal cord using stem cells found in bone marrow.

The treatment was approved for use on the NHS in Scotland in 2019 but the criteria is so tight that even Jodie, with her frequent relapses, doesn't qualify for it.

Her only option has been to go private abroad - at a cost of 50,000 - and her sister Tricia has launched a GoFundMe page to crowdfund the costs of the operation.

Since publishing the page last week, over 13,000 has been donated by well-wishers, giving Jodie hope she can put a halt to her body's war against itself.

HSCT won't fix the damage done to nerves nerves in the last decade, and is not without its risks, with side-effects such as increased risk of developing cancer.

However, it should put a stop to further degeneration - and serves as Jodie's last hope to live a life relatively free of MS.

"Every relapse is like setting my body up from scratch, and it happens again and again," she added.

"If my MS becomes progressive there's not a lot of treatment available for that all.

"I know from my own research it's beneficial doing the treatment sooner rather than later - and I'm too young not to try it now.

"My next relapse will probably put me in a wheelchair. But you wouldn't look at me 90% of the time and think there's anything wrong.

"To be honest, I feel like this is the last hope. This is the most extreme treatment you can get - it's chemotherapy.

"There's not much else I can do after this but it can't wait.

"It won't be an easy fix, it's not a bounce back - but it will stop the progression, and that's the goal.

"I want to be able to live my life - that's all I ask."

Jodie's sister Tricia Moran, who spearheaded the fundraising appeal, said: "Watching Jodie go through that first episode...it was quite heartbreaking as a family to watch.

"We didn't get any answers for a long time and it really impacted on her confidence - we couldn't reassure her and felt quite helpless.

"As a family we've seen her struggle so much with her relapses and she's so aware of what she's lost.

"She can't just take Ethan to the toy shop on a whim - everything has to be planned.

"It's an understatement to say how overwhelmed we have been by the kindness of friends, family and complete strangers."

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Scots mum with MS says 50k treatment abroad is 'last hope' of halting disease - Daily Record

Spinal Cord Stem Cells Comments Off on Scots mum with MS says 50k treatment abroad is ‘last hope’ of halting disease – Daily Record | January 18th, 2022

Spinal cord injuries can be devastating and there are currentlyfew options to reverse the effects, which can include paralysis, chronic pain and loss of bladder control.

But an international team of researchers, including the University of Torontos Molly Shoichet,hopes to change that.

Over the past few years, weve made a lot of progress in tissue engineering, drug delivery and regenerative medicine, says Shoichet, a University Professor in the department of chemical engineering and applied chemistry in the Faculty of Applied Science & Engineering, the Institute of Biomedical Engineering and the Donnelly Centre for Cellular and Biomolecular Research.

With this ambitious project, we bring world leading experts together to try to do something that no one else has been able to do: promote repair and regeneration in the injured spinal cord.

Shoichet is a co-principal investigator withMend the Gap, an international collaboration of more than 30 researchers, engineers, scientists, surgeons and social scientists from Canada, the United States, Europe and Australia. The collaboration this week received $24 million from Canadas New Frontiers in Research Fund to advance their work.

The team takes its name from the fact that only a small gap, just a few centimetres long, is responsible for blocking the nerve impulses that normally flow through the spinal cord. Bridging that gap requires collaboration from some of the worlds top experts in a wide range of fields.

Shoichet is known internationally for her work on hydrogels biocompatible materials that can help facilitate tissue repair. Hydrogels can function as scaffolds, enhancing or augmenting natural processes that serve to repair damaged tissue.

Hydrogels can also serve as controlled-release mechanisms for drugs that aid healing, or to protect stem cells that are being injected into the body bykeeping them alive and healthy while they integrate into damaged tissues.

Another important line of research involves dealing with the glial scar that forms in the wake of a spinal cord injury. In the short term, this protective shield of cells and biochemicals prevents further injury in the damaged nerve, but in the long termit can serve as a barrier to nerve repair.

Shoichet and her team bring their expertise in hydrogels and local delivery strategies to deliver innovative biomolecules locally and directly to the injured spinal cord. For example, shere-engineered an enzymeto selectively degrade some of the biomolecules that make up the glial scar. This redesigned enzyme is both more stable and more active than the wild type.

By breaking through the glial scar with this new delivery strategy, the enzyme can enable other therapies from advanced drugs to stem cells to further promote tissue regeneration and repair.

The environment in the injured spinal cord is a very complicated place, says Shoichet. There are a whole range of natural processes at work some of which we want to enhance, others of which we need to find ways to circumvent. I am very excited to be part of this multidisciplinary team, which has the breadth and depth of expertise that we need to make a real difference when it comes to treating spinal cord injury.

Shoichet is the only person to be elected a fellow of all three of Canadas national academiesand is a foreign member of the U.S. National Academy of Engineering and a fellow of the Royal Society of London. She was the 2020 recipient of the Gerhard Herzberg Canada Gold Medal, Canadas highest honour for science and engineering research. She is also a member of the Order of Ontario and an Officer of the Order of Canada.

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Mending the gap: U of T's Molly Shoichet joins team developing new treatments for spinal cord injuries - News@UofT

Spinal Cord Stem Cells Comments Off on Mending the gap: U of T’s Molly Shoichet joins team developing new treatments for spinal cord injuries – News@UofT | January 18th, 2022

Definition

A spinal cord injury (SCI) is damage to the tight bundle of cells and nerves that sends and receives signals from the brain to and from the rest of the body. SCI can be caused by direct injury to the spinal cord itself or from damage to the tissue and bones (vertebrae) that surround the spinal cord. This damage can result in temporary or permanent changes in sensation, movement, strength, and body functions below the site of injury. Some injuries that cause little or no cell death may allow for an almost complete recovery while those that occur higher on the spinal cord and are more serious can cause paralysis in most of the body. Motor vehicle accidents and catastrophic falls are the most common causes of SCI in the United States.

An incomplete injury means the spinal cord is still able to trasnmit some messages to and from the brain to the rest of the body. A complete injury means there is no nerve communication and motor function (voluntary movement) below the site where the trauma occurred.

A spinal cord injury can cause one or more symptoms including:

Definition

A spinal cord injury (SCI) is damage to the tight bundle of cells and nerves that sends and receives signals from the brain to and from the rest of the body. SCI can be caused by direct injury to the spinal cord itself or from damage to the tissue and bones (vertebrae) that surround the spinal cord. This damage can result in temporary or permanent changes in sensation, movement, strength, and body functions below the site of injury. Some injuries that cause little or no cell death may allow for an almost complete recovery while those that occur higher on the spinal cord and are more serious can cause paralysis in most of the body. Motor vehicle accidents and catastrophic falls are the most common causes of SCI in the United States.

An incomplete injury means the spinal cord is still able to trasnmit some messages to and from the brain to the rest of the body. A complete injury means there is no nerve communication and motor function (voluntary movement) below the site where the trauma occurred.

A spinal cord injury can cause one or more symptoms including:

Treatment

Immediate treatment at the accident scene includes putting the person on a backboard with a special collar around the neck to prevent further damage to the spinal cord. Treatment at a trauma center may include realigning the spine and surgery to remove any bone fragments or other objects that might press on the spinal column.

Rehabilitative care may include breathing assistance using a machine that produces forced air, treatment for any respiratory or circulatory problems, pain medications, and learning new ways to address bladder and bowel problems. A rehabilitation team will assess the individual's needs and create a rehabilitation program that combines plysical and other therapies with skill-building activities, training, and counseling to aid recovery and provide social and emotional support, as well as to increase independence and quality of life.

Treatment

Immediate treatment at the accident scene includes putting the person on a backboard with a special collar around the neck to prevent further damage to the spinal cord. Treatment at a trauma center may include realigning the spine and surgery to remove any bone fragments or other objects that might press on the spinal column.

Rehabilitative care may include breathing assistance using a machine that produces forced air, treatment for any respiratory or circulatory problems, pain medications, and learning new ways to address bladder and bowel problems. A rehabilitation team will assess the individual's needs and create a rehabilitation program that combines plysical and other therapies with skill-building activities, training, and counseling to aid recovery and provide social and emotional support, as well as to increase independence and quality of life.

Definition

A spinal cord injury (SCI) is damage to the tight bundle of cells and nerves that sends and receives signals from the brain to and from the rest of the body. SCI can be caused by direct injury to the spinal cord itself or from damage to the tissue and bones (vertebrae) that surround the spinal cord. This damage can result in temporary or permanent changes in sensation, movement, strength, and body functions below the site of injury. Some injuries that cause little or no cell death may allow for an almost complete recovery while those that occur higher on the spinal cord and are more serious can cause paralysis in most of the body. Motor vehicle accidents and catastrophic falls are the most common causes of SCI in the United States.

An incomplete injury means the spinal cord is still able to trasnmit some messages to and from the brain to the rest of the body. A complete injury means there is no nerve communication and motor function (voluntary movement) below the site where the trauma occurred.

A spinal cord injury can cause one or more symptoms including:

Treatment

Immediate treatment at the accident scene includes putting the person on a backboard with a special collar around the neck to prevent further damage to the spinal cord. Treatment at a trauma center may include realigning the spine and surgery to remove any bone fragments or other objects that might press on the spinal column.

Rehabilitative care may include breathing assistance using a machine that produces forced air, treatment for any respiratory or circulatory problems, pain medications, and learning new ways to address bladder and bowel problems. A rehabilitation team will assess the individual's needs and create a rehabilitation program that combines plysical and other therapies with skill-building activities, training, and counseling to aid recovery and provide social and emotional support, as well as to increase independence and quality of life.

Prognosis

Retention of movement depends on the type of injury and where it occurs along the spine. Loss of nerve function occurs below the level of injury. An injury higher on the spinal cord can cause paralysis in most of the body and affect all limbs (called tetraplegia or quadriplegia). A lower injury to the spinal cord may cause paralysis affecting the legs and lower body (called paraplegia).

People who survive a spinal cord injury will most likely have medical complications such as chronic pain and bladder and bowel dysfunction, along with an increased susceptibility to respiratory and heart problems. Successful recovery depends upon how well these chronic conditions are handled day to day.

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Prognosis

Retention of movement depends on the type of injury and where it occurs along the spine. Loss of nerve function occurs below the level of injury. An injury higher on the spinal cord can cause paralysis in most of the body and affect all limbs (called tetraplegia or quadriplegia). A lower injury to the spinal cord may cause paralysis affecting the legs and lower body (called paraplegia).

People who survive a spinal cord injury will most likely have medical complications such as chronic pain and bladder and bowel dysfunction, along with an increased susceptibility to respiratory and heart problems. Successful recovery depends upon how well these chronic conditions are handled day to day.

Prognosis

Retention of movement depends on the type of injury and where it occurs along the spine. Loss of nerve function occurs below the level of injury. An injury higher on the spinal cord can cause paralysis in most of the body and affect all limbs (called tetraplegia or quadriplegia). A lower injury to the spinal cord may cause paralysis affecting the legs and lower body (called paraplegia).

People who survive a spinal cord injury will most likely have medical complications such as chronic pain and bladder and bowel dysfunction, along with an increased susceptibility to respiratory and heart problems. Successful recovery depends upon how well these chronic conditions are handled day to day.

Definition

A spinal cord injury (SCI) is damage to the tight bundle of cells and nerves that sends and receives signals from the brain to and from the rest of the body. SCI can be caused by direct injury to the spinal cord itself or from damage to the tissue and bones (vertebrae) that surround the spinal cord. This damage can result in temporary or permanent changes in sensation, movement, strength, and body functions below the site of injury. Some injuries that cause little or no cell death may allow for an almost complete recovery while those that occur higher on the spinal cord and are more serious can cause paralysis in most of the body. Motor vehicle accidents and catastrophic falls are the most common causes of SCI in the United States.

An incomplete injury means the spinal cord is still able to trasnmit some messages to and from the brain to the rest of the body. A complete injury means there is no nerve communication and motor function (voluntary movement) below the site where the trauma occurred.

A spinal cord injury can cause one or more symptoms including:

Treatment

Immediate treatment at the accident scene includes putting the person on a backboard with a special collar around the neck to prevent further damage to the spinal cord. Treatment at a trauma center may include realigning the spine and surgery to remove any bone fragments or other objects that might press on the spinal column.

Rehabilitative care may include breathing assistance using a machine that produces forced air, treatment for any respiratory or circulatory problems, pain medications, and learning new ways to address bladder and bowel problems. A rehabilitation team will assess the individual's needs and create a rehabilitation program that combines plysical and other therapies with skill-building activities, training, and counseling to aid recovery and provide social and emotional support, as well as to increase independence and quality of life.

Prognosis

Retention of movement depends on the type of injury and where it occurs along the spine. Loss of nerve function occurs below the level of injury. An injury higher on the spinal cord can cause paralysis in most of the body and affect all limbs (called tetraplegia or quadriplegia). A lower injury to the spinal cord may cause paralysis affecting the legs and lower body (called paraplegia).

People who survive a spinal cord injury will most likely have medical complications such as chronic pain and bladder and bowel dysfunction, along with an increased susceptibility to respiratory and heart problems. Successful recovery depends upon how well these chronic conditions are handled day to day.

What research is being done?

Scientists at the National Institute of Neurological Disorders and Stroke (NINDS) and those at other institutes at the National Institutes of Health (NIH) conduct and fund research to better understand SCI and how to treat it.

Current research on SCI focuses on advancing our understanding of four key principles of spinal cord repair:

Basic spinal cord function research studies how the normal spinal cord develops, processes sensory information, controls movement, and generates rhythmic patterns (like walking and breathing). Research on injury mechanisms focuses on what causes immediate harm and on the cascade of helpful and harmful bodily reactions that protect from or contribute to damage in the hours and days following a spinal cord injury. Neural engineering strategies also offer ways to restore communication and independence.

Information from the National Library of Medicines MedlinePlusSpinal Cord Injuries

Patient Organizations

Christopher and Dana Reeve Foundation

636 Morris Turnpike

Suite 3A

Short Hills

NJ

Short Hills, NJ 07078

Tel: 973-379-2690; 800-225-0292

Miami Project to Cure Paralysis

1095 NW 14th Terrace

Lois Pope LIFE Center

Miami

FL

Miami, FL 33136

Tel: 305-243-6001; 800-STANDUP (782-6387)

National Institute on Disability, Independent Living, and Rehabilitation Research (NIDILRR)

Administration for Community Living

330 C St., NW

Washington

DC

Washington, DC 20201

Tel: 202-401-4634; 202-245-7316 (TTY)

National Rehabilitation Information Center (NARIC)

8400 Corporate Drive

Suite 500

Landover

MD

Landover, MD 20785

Tel: 301-459-5900; 800-346-2742; 301-459-5984 (TTY)

National Spinal Cord Injury Statistical Center

1717 6th Avenue South

Birmingham

AL

Birmingham, AL 35232

Paralyzed Veterans of America (PVA)

801 18th Street, NW

Washington

DC

Washington, DC 20006-3517

Tel: 800-424-8200

United Spinal Association

120-34 Queens Boulevard, #320

Kew Gardens

NY

Kew Gardens, NY 11415

Tel: 718-803-3782; 800-962-9629

Publications

Spasticity information sheet compiled by NINDS, the National Institute of Neurological Disorders and Stroke.

Myoclonus fact sheet compiled by the National Institute of Neurological Disorders and Stroke (NINDS).

Patient Organizations

Christopher and Dana Reeve Foundation

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Spinal Cord Injury Information Page | National Institute ...

Spinal Cord Stem Cells Comments Off on Spinal Cord Injury Information Page | National Institute … | January 3rd, 2022

After the therapy performs its function, the materials biodegrade into nutrients for the cells within 12 weeks and then completely disappear from the body without noticeable side effects.This is the first study in which researchers controlled the collective motion of molecules through changes in chemical structure to increase a therapeutics efficacy.

Samuel I. Stupp

Our research aims to find a therapy that can prevent individuals from becoming paralyzed after major trauma or disease, said NorthwesternsSamuel I. Stupp, who led the study. For decades, this has remained a major challenge for scientists because our bodys central nervous system, which includes the brain and spinal cord, does not have any significant capacity to repair itself after injury or after the onset of a degenerative disease. We are going straight to the FDA to start the process of getting this new therapy approved for use in human patients, who currently have very few treatment options.

Stupp is Board of Trustees Professor of Materials Science and Engineering, Chemistry, Medicine and Biomedical Engineering at Northwestern, where he is founding director of theSimpson Querrey Institute for BioNanotechnology(SQI) and its affiliated research center, theCenter for Regenerative Nanomedicine. He has appointments in theMcCormick School of Engineering,Weinberg College of Arts and SciencesandFeinberg School of Medicine.

According to the National Spinal Cord Injury Statistical Center, nearly 300,000 people are currently living with a spinal cord injury in the United States. Life for these patients can be extraordinarily difficult. Less than 3% of people with complete injury ever recover basic physical functions. And approximately 30% are re-hospitalized at least once during any given year after the initial injury, costing millions of dollars in average lifetime health care costs per patient. Life expectancy for people with spinal cord injuries is significantly lower than people without spinal cord injuries and has not improved since the 1980s.

I wanted to make a difference on the outcomes of spinal cord injury and to tackle this problem, given the tremendous impact it could have on the lives of patients.

Currently, there are no therapeutics that trigger spinal cord regeneration, said Stupp, an expert in regenerative medicine. I wanted to make a difference on the outcomes of spinal cord injury and to tackle this problem, given the tremendous impact it could have on the lives of patients. Also, new science to address spinal cord injury could have impact on strategies for neurodegenerative diseases and stroke.

A new injectable therapy forms nanofibers with two different bioactive signals (green and orange) that communicate with cells to initiate repair of the injured spinal cord. Illustration by Mark Seniw

The secret behind Stupps new breakthrough therapeutic is tuning the motion of molecules, so they can find and properly engage constantly moving cellular receptors. Injected as a liquid, the therapy immediately gels into a complex network of nanofibers that mimic the extracellular matrix of the spinal cord. By matching the matrixs structure, mimicking the motion of biological molecules and incorporating signals for receptors, the synthetic materials are able to communicate with cells.

Receptors in neurons and other cells constantly move around, Stupp said. The key innovation in our research, which has never been done before, is to control the collective motion of more than 100,000 molecules within our nanofibers. By making the molecules move, dance or even leap temporarily out of these structures, known as supramolecular polymers, they are able to connect more effectively with receptors.

100,000molecules move within the nanofibers

Stupp and his team found that fine-tuning the molecules motion within the nanofiber network to make them more agile resulted in greater therapeutic efficacy in paralyzed mice. They also confirmed that formulations of their therapy with enhanced molecular motion performed better during in vitro tests with human cells, indicating increased bioactivity and cellular signaling.

Given that cells themselves and their receptors are in constant motion, you can imagine that molecules moving more rapidly would encounter these receptors more often, Stupp said. If the molecules are sluggish and not as social, they may never come into contact with the cells.

Once connected to the receptors, the moving molecules trigger two cascading signals, both of which are critical to spinal cord repair. One signal prompts the long tails of neurons in the spinal cord, called axons, to regenerate. Similar to electrical cables, axons send signals between the brain and the rest of the body. Severing or damaging axons can result in the loss of feeling in the body or even paralysis. Repairing axons, on the other hand, increases communication between the body and brain.

Zaida lvarez

The second signal helps neurons survive after injury because it causes other cell types to proliferate, promoting the regrowth of lost blood vessels that feed neurons and critical cells for tissue repair. The therapy also induces myelin to rebuild around axons and reduces glial scarring, which acts as a physical barrier that prevents the spinal cord from healing.

The signals used in the study mimic the natural proteins that are needed to induce the desired biological responses. However, proteins have extremely short half-lives and are expensive to produce, said Zaida lvarez, the studys first author. Our synthetic signals are short, modified peptides that when bonded together by the thousands will survive for weeks to deliver bioactivity. The end result is a therapy that is less expensive to produce and lasts much longer.

A former research assistant professor in Stupps laboratory,lvarez is now a visiting scholar at SQI and a researcher at theInstitute for Bioengineering of Catalonain Spain.

While the new therapy could be used to prevent paralysis after major trauma (automobile accidents, falls, sports accidents and gunshot wounds) as well as from diseases, Stupp believes the underlying discovery that supramolecular motion is a key factor in bioactivity can be applied to other therapies and targets.

The central nervous system tissues we have successfully regenerated in the injured spinal cord are similar to those in the brain affected by stroke and neurodegenerative diseases, such as ALS, Parkinsons disease and Alzheimers disease, Stupp said. Beyond that, our fundamental discovery about controlling the motion of molecular assemblies to enhance cell signaling could be applied universally across biomedical targets.

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