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

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

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

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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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Whether a deadly disease like cancer and Alzheimers or a lifelong affliction like diabetes, eczema, or arthritis, 2021 has been a year of breakthroughs and advancements.

Beyond COVID-19 and the developments of the mRNA vaccines created to halt the pandemic, medical researchers around the world continued to focus on the long-entrenched problems that have plagued our health for centuries.

Here are some of the top Health stories from 2021:

Routinely polled as one of the most-feared diseases, Alzheimers researchers have hailed several achievements this year.

One fascinating focus has been on prevention, or what contributes to the disease.

A neuroscientist who authored a book called The First Survivors of Alzheimers is not so much focused on drugs as he is focused on brain prevention and is achieving results never before seen in the history of Alzheimers treatment. (Read more)

The findings of a drug that seemed to restore normal cognition in a variety of cases ranging from traumatic brain injury, to noise-related hearing loss, to neurodegenerative disease seem to suggest, its creators write, that age-related cognitive loss may be down to a physiological blockage rather than permanent damage. (Read More)

As seen many times before, sometimes the best new cure is an old drug. Four drugstwo non-steroidal anti-inflammatories, along with two anti-hypertensives, proved effective at reversing Alzheimers disease and neutralizing symptoms in mice suffering from various stages of the illness. (Read More)

As long as theres lifeforms, there will be cancer, but that doesnt mean we cant learn how to treat it, strike at the root cause, and hopefully turn at least some forms of it from one of the major killers to a minor inconvenience.

With 12,000 Britons diagnosed with head and neck cancer every year, the results of a phase III trial that saw complete eradication in some patients, and side-effect-free life extension in others, has the country excited. (Read More)

Discovering an RNA molecule that regulates a key driver in the growth of prostate cancer cells is noteworthy because prostate cancer is one of the most common in men around the world, and because most drugs work for a short period of time before the cancer becomes resistant to it. (Read More)

Despite the gradual awareness of the harmful effects of sugar and bread on the body, chronic diabetes and juvenile diabetes continues to be a major problem in our society.

It turns out that all it takes for this potential cure to rid a patient of a debilitating autoimmune disease is a small piece of adult skin no larger than a housefly. With FDA trials underway, hundreds of thousands of Type-1 diabetics have a chance at a potential cure. (Read More)

Nearly 500 million diabetics around the world need to mildly stab themselves in order to ensure they are in no danger of going into shock. An Australian med-tech company has a new solution. (Read More)

Afflicting a quarter of all Americans, and the leading cause of workplace disability resulting in $303 billion in lost productivity, arthritis took a step towards a cure in 2021.

An alternative to highly addictive painkillers is offering those who undergo knee replacements a large measure of safe relief. Many arthritis patients have knees and hips replaced in the hope of regaining some measure of mobility later in life, but the resulting pain and stiffness can sometimes only be treated with opioids. (Read More)

Osteoarthritis is the most common form, and it affects 8.5 million people. Nasal cells come from a special class of adaptive tissues produced in the brain and spinal cord that can be used to relieve chronic inflammation in the knee and lay the groundwork for a therapeutic treatment that spares patients of surgery and prosthesis. (Read More)

It would seem silly to write a list such as this without addressing the elephant in the room, but as the pandemic petered on through 2021, breakthroughs continued to be made.

One of Americas most favorite medicines was found, unsurprisingly to some doctors, to have as strong an effect as vaccines in some cases at mitigating the severe symptoms of COVID-19. (Read More)

Along with an Israeli nasal spray that prevented infection in 99% of patients, another was found in trials at the University of Oxford which killed 99% of the virus in the nasal passage. (Read More)

Some demonstrations of prosthetic internal organs have shocked the world in 2021, providing a glimpse of a sci-fi future for human anatomy.

A bio-tech implant that allowed a 78-year old blind man to see his family again actually binds with the inside of the eye-socket in a way that had never been done before. (Read More)

The worlds first legit prototype for an artificial kidney was successfully tested when the blood filter and bio reactor components were demonstrated to work together, offering hope to free kidney disease patients from dialysis machines and transplant lists. (Read More)

Ticks, as awful as they are, have their place in the Web of Life. Researchers have identified a soil microbe that eliminates Lyme Disease but essentially nothing else, not even the ticks, opening the door to ecosystem wide treatment against Lyme Disease. (Read More)

Stem cells prepared with the patients own bone marrow were used to repair damaged spinal cords and restore mobility and motor functions in more than half of a Yale scientists trial. (Read More)

An incurable autoimmune disorder that results in progressive motor function loss and neurodegeneration, an MS breakthrough was achieved using the same mRNA vaccines that worked so well originally to stop the COVID pandemic. (Read More)

A monoclonal antibody that reduces the amount of inflammatory molecules that cause a hormonal dysregulation leading to eczema was a treatment generated by this totally surprise finding. (Read More)

Habit Cough the name for a cough without a cause has been cured through a YouTube video relying mostly on the power of suggestion. While this may seem a little sketchy, many people with habit cough have no underlying respiratory condition of any kind, and therefore an ounce of suggestion may beat a cure. (Read More)

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Best 2021 Medical Breakthroughs And Treatments to Beat Cancer, Alzheimer's, Diabetes & More - Good News Network

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DUBLIN--(BUSINESS WIRE)--The "Global Regenerative Medicine Market Size, Share & Trends Analysis Report by Product (Cell-based Immunotherapies, Gene Therapies), by Therapeutic Category (Cardiovascular, Oncology), and Segment Forecasts, 2021-2027" report has been added to ResearchAndMarkets.com's offering.

The global regenerative medicine market size is expected to reach USD 57.08 billion by 2027, growing at a CAGR of 11.27% over the forecast period.

Recent advancements in biological therapies have resulted in a gradual shift in preference toward personalized medicinal strategies over the conventional treatment approach. This has resulted in rising R&D activities in the regenerative medicine arena for the development of novel regenerative therapies.

Furthermore, advancements in cell biology, genomics research, and gene-editing technology are anticipated to fuel the growth of the industry. Stem cell-based regenerative therapies are in clinical trials, which may help restore damaged specialized cells in many serious and fatal diseases, such as cancer, Alzheimer's, neurodegenerative diseases, and spinal cord injuries.

For instance, various research institutes have adopted Human Embryonic Stem Cells (hESCs) to develop a treatment for Age-related Macular Degeneration (AMD).

Constant advancements in molecular medicines have led to the development of gene-based therapy, which utilizes targeted delivery of DNA as a medicine to fight against various disorders.

Gene therapy developments are high in oncology due to the rising prevalence and genetically driven pathophysiology of cancer. The steady commercial success of gene therapies is expected to accelerate the growth of the global market over the forecast period.

Regenerative Medicine Market Report Highlights

Key Topics Covered:

Market Variables, Trends, & Scope

Competitive Analysis

Covid-19 Impact Analysis

Regenerative Medicine Market: Product Business Analysis

Regenerative Medicine Market: Therapeutic Category Business Analysis

Regenerative Medicine Market: Regional Business Analysis

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/kovhgl

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Global Regenerative Medicine Market is Expected to Reach USD 57.08 Billion by 2027, Growing at a CAGR of 11.27% Over the Forecast Period. -...

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Image by MasterTux from Pixabay

The brain is the most complex thing in the universe! It is made up of an intricate network of cells called neurons. Neurons are long, elongated, fibre-like cells, and billions of them form a complex network of connections called synapses. Neurons are not physically connected, but they transmit messages between them electrochemically as non-contact nerve impulses. And, there are trillions of such connections in our brain. However, in the initial stages, when the embryo is developing, the primitive neurons are rounded and lack connection to each other.

So how do these innocuous-looking rounded cells become highly connected elongated neurons? Researchers from Manipal Institute of Regenerative Medicine, Bengaluru, found the key gene that assists in making this happen. The study published in iScience journal shows that a gene called Superoxide dismutase 2 (SOD2), hitherto known to be involved in another function, is caught performing a completely different function -- promoting the generation of neurons. The authors state that although a complete understanding of the exact mechanism of how this happens remains to be unravelled, there is a possibility that one day, human nerve cells could be grown from any human tissue cells, thereby opening therapeutic avenues for patients with nerve or spinal cord injuries.

Existing literature indicates that SOD2 basically mops oxygen radicals inside the cell. During normal metabolism, cell components called mitochondria generate energy-rich molecules from carbon sources. However, the process produces an undesirable byproduct called oxygen radicals. These are oxygen molecules with an extra electron on them which makes them highly reactive with other molecules, thereby causing toxicity in the cells. The usually designated job of the SOD2 gene is to minimise this damage by mopping up these free oxygen radicals. The researchers found that the SOD2 mop had another function: to help the cells become neural precursors, which in turn become highly connected neurons. The process is termed Differentiation.

Scientists differentiate a neuron cell from an embryonic cell by its shape and by looking for specific proteins produced only in these neuronal cells. These proteins are markers for that particular cell type.

To decipher SOD2s role in the differentiation process, the researchers introduced copies of the SOD2 gene into mouse embryonic stem cells grown in the lab (cultured cells). When they increased the number of copies of the gene, the embryonic cells changed into a neuron-like appearance and exhibited markers unique to cells of neurons. However, the markers were absent when they eliminated the SOD2 gene.

In our study, using embryonic cells, we show that when SOD2 is knocked down or eliminated and subjected to differentiation, the embryonic cells could not specifically change into a neuron. However, this did not compromise the differentiation to other tissues, says Dr Anujith Kumar, corresponding author of the paper.

Owing to numerous ethical problems associated with procuring human embryonic cells, the researchers used fibroblasts or skin cells of mice and intended to convert them into stem cells that mimic embryonic cells. They achieved this by introducing another gene called OCT4 into the fibroblast cells. When the researchers transferred the SOD2 gene and OCT4, fibroblasts stopped being fibroblasts and changed into neurons, but not pluripotent stem cells. (Pluripotent stem cells are master cells that can differentiate into almost any tissue cell type).

So how does SOD2 actually do this? The researchers hypothesised that SOD2 could be having other functions that involved mitochondria. However, they had to first observe the microscopic mitochondria inside the cell to test their hypothesis. To do so, they tagged a protein found on the mitochondrial surface with a fluorescent dye. Under a fluorescent microscope, these tagged mitochondria appear fluorescent. When the SOD2 gene was introduced in the cell, they could see that the mitochondria were longer than they would be. This is because the individual mitochondria had fused to produce longer filament like mitochondria.

Mitochondria fuse because of a protein called MFN2. Researchers found that the expression of SOD2 was causing the overproduction of MFN2 protein. The fusing of mitochondria was somehow related to the embryonic cells elongating and growing into neurons. But how exactly that happens is still a mystery.

Mechanistically, it is unclear how mitochondrial fusion and fission favour commitment to neuron formation, says Dr Kumar. However, he speculates that As neurons are dynamic cells and dependent on excessive energy molecule ATP (adenosine tri-phosphate), probably mitochondrial fusion favours the energy supply and in turn facilitates neural formation.

The research done on mouse cells needs to be repeated with human cells, and hopefully, the results will one day be helpful to treat nerve injuries. At this juncture, the current findings on differentiated neurons thus produced are suitable for research purposes to study neuronal development. It could also be used to develop an experimental framework to model diseases in the cells by growing them in the lab. Such experiments could be utilised for drug screening and also where researchers test the effect of promising drugs by trying them on these cells.

This article has been run past the researchers, whose work is covered, to ensure accuracy.

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Researchers globally produce hundreds of thousands of studies annually. It can be difficult to know if at some time in the future they will be the foundation for a disease cure or a technology such as CRISPR that revolutionizes medicine. But many are exciting for what they point to or how they spike the imagination. Heres a look at 10 of the more compelling research stories of the year.

Type 1 Diabetes Therapy Showed Promise in Early-Stage Trial

Vertex Pharmaceuticalsannouncedpositive early data from the first patient in its Phase I/II study of VX-880 in type 1 diabetes (T1D). The therapy is a stem cell-derived, fully differentiated pancreatic islet cell replacement therapy. T1D is an autoimmune disease, where the immune system attacks the islet cells in the pancreas, which is where insulin is produced. This leads to loss of insulin production and problems with blood sugar control.

In the study, the patient received a single infusion of VX-880 at half the target dose along with immunosuppressive therapy. The patient showed successful engraftment and demonstrated fast and robust improvements in several measurements, including increases in fasting and stimulated C-peptide, improvements in glycemic control, including HbA1c. It also resulted in less need for medical insulin. The therapy appeared well tolerated.

Some Alzheimers Plaques May Be Protective

Source: BioSpace

One of the hallmarks of Alzheimers disease is the buildup of beta-amyloid plaques in the brain. Yet many drugs that cleared amyloid dont seem to improve memory or cognition. Many researchers believe amyloid is only part of the issue, perhaps triggering inflammation that causes damage to the brain. New research out of theSalk Instituteadded a new twist, suggesting that some of the plaques may be protective. A type of immune cell in the brain, microglia, was long believed to inhibit the growth of plaques by eating them. Their research, however, demonstrated that microglia promote the formation of what are being dubbed dense-core plaques, which transports the wispy plaque away from neurons. They published their research in the journalNature Immunology.

We show that dense-core plaques dont form spontaneously, said Greg Lemke, a professor in Salks Molecular Neurobiology Laboratory. We believe theyre built by microglia as a defense mechanism, so they may be best left alone. There are various efforts to get the FDA to approve antibodies whose main clinical effect is reducing dense-core plaque formation, but we make the argument that breaking up the plaque may be doing more damage.

5 Genes Associated with Lewy Body Dementia, with Implications for Alzheimers and Parkinsons

Research conductedby theNIHs National Institute of Neurological Disorders and Stroke (NINDS)identified five genes that appear to play a critical role in whether a person will suffer from Lewy body dementia, a type of dementia where the brain accumulates clumps of abnormal protein deposits known as Lewy bodies. The data also supported Lewy body dementias connections to Parkinsons disease and connections to Alzheimers disease. The research was published in the journalNature Genetics.

Sonja Scholz, investigator at the NIHs NINDS and senior author of the study, said, Our results support the idea that this may be because Lewy body dementia is caused by a spectrum of problems that can be seen in both disorders. We hope that these results will act as a blueprint for understanding the disease and developing new treatments.

Why Obesity is Associated with Inflammation

Although obesity is linked with many inflammatory conditions, including cancer, diabetes, heart disease, and infection, why isnt it well understood? Researchers atUT Southwestern Medical Centeridentifieda type of cell that, at least in mice, is responsible for triggering inflammation in fat tissue. In obese individuals, white adipose tissue (WAT), stores excess calories in the form of triglycerides. In obesity, WAT is overworked, fat cells start to die, and immune cells are activated. The research team identified an adipose progenitor cell (APC), a precursor that later generates mature fat cells. These new cells are called fibro-inflammatory progenitors (FIPs) and they make signals that encourage inflammation.

Whats Behind Brain Fog in COVID-19 Patients

One of several unusual symptoms reported in COVID-19 patients is what is dubbed brain fog or COVID brain, but in medical terminology, is called encephalopathy. It appears to be loss of short-term memory, headaches and confusion. At its most severe, it is associated with psychosis and seizures. Researchers atMemorial Sloan Kettering Cancer Centerpublishedresearch in the journalCancer Cellthat explains the underlying cause of brain fog.

Jan Remsik, a research fellow in the lab, says, We found that these patients had persistent inflammation and high levels of cytokines in their cerebrospinal fluid, which explained the symptoms they were having.

New Compound Appears to Reverse Neuron Damage Caused by ALS

Researchers atNorthwestern Universityidentifieda compound that appears to reverse the ongoing degeneration of upper motor neurons associated with amyotrophic lateral sclerosis (ALS). ALS is a progressive neurodegenerative disease affecting nerve cells in the brain and spinal cord. As the motor neurons degenerate, they eventually die and the ability of the brain to initiate and control muscle movement is lost. With the disease, people may lose the ability to speak, eat, move and breathe. The compound, NU-9, was developed in the laboratory of Richard B. Silverman, the Patrick G. Ryan/Aon Professor of Chemistry at Northwestern. It can reduce protein misfolding in critical cell lines. The compound is also not toxic and can cross the blood-brain barrier. They published their research inClinical and Translational Medicine.

How Astrocytes Fix Damage in the Brain

Investigators withCharit Universittsmedizin Berlindescribed how a type of glial cell, called astrocytes, plays a role in protecting surrounding brain tissue after damage. They become part of a defense mechanism called reactive astrogliosis, which helps form scars, and contains inflammation and controls tissue damage. Astrocytes are also able to ensure the nerve cells survive that are located immediately next to the tissue injury, which preserves the function of neuronal networks. The mechanism was the protein drebrin, which controls astrogliosis. Astrocytes require drebrin to form scars and protect the surrounding tissue. Drebrin regulates the reorganization of the actin cytoskeleton, an internal scaffold that maintains astrocyte mechanical stability.

A New Spin on Jurassic Park?

In the books and filmsJurassic Park, researchers collected the blood from insects trapped in amber and cloned dinosaurs. A researcher from theUniversity of Minnesota is putting a more practical spin on amber research. Amber is the fossilized resin from a now-extinct species of pine, Sciadopityaceae. It was formed about 44 million years ago. In the Baltic regions, amber has been used for hundreds of years for traditional medicines for pain relief and its anti-inflammatory and anti-infective properties. Previous research has suggested that amber molecules might have an antibiotic effect. The team extracted even more chemicals from amber samples that appeared to show activity against gram-positive, antibiotic resistant bacteria.

They identified 20 compounds using GC-MS in the amber, most prominent being abietic acid, dehydroabietic acid and palustric acid, compounds with known biological activity. They also acquired a Japanese umbrella pine, the closest living species to theSciadopityaceae, and extracted resins and identified sclarene, a molecule present in the amber extracts that could potentially undergo chemical transformations to produce the bioactive molecules found in the Baltic amber samples.

The most important finding is that these compounds are active against gram-positive bacteria, such as certain Staphylococcus aureus strains, but not gram-negative bacteria, said Connor McDermott, a graduate student in the laboratory of Elizabeth Ambrose, who led the research. This implies the composition of the bacterial membrane is important for the activity of the compounds.

Genetics of People Who Live 105 or Older

A new study of 81 semi-supercentenarianspeople 105 years of age or olderand supercentenarians110 years or older from across Italy, werestudiedby researchers from theUniversity of Bologna, Italy andNestle Research in Lausanne, Switzerland. They compared genetic data from these extraordinary agers to 36 healthy people from the same region whose age, on average, was 68 years. Blood samples were drawn, and whole-genome sequencing was performed. They then compared their data with another previously published study that analyzed 333 Italians over 100 years of age and 358 people who were about 60 years of age. They published their research in the journal eLife.

Scientists identified five common genetic changes that were most frequent in the 105+/110+ groups, between two genes known as COA1 and STK17A. Analysis showed the same variants in the people over 100. Computational analysis predicted these variations most likely modulated the expression of three different genes: STK17A, COA1 and BLVRA.

Junk DNA and Aging

For a long time, so-called junk DNA was thought to play no role in inheritance or metabolism. Increasingly, this non-coding DNA is found to play a significant role in gene regulation. Researchers atWashington State Universityrecently identifieda DNA region called VNTR2-1 that seems to drive telomerase gene activity. In addition, it appears to prevent aging in some types of cells. Telomeres are the ends of chromosomes, and their length is associated with aging that is to say, as the older you get, the shorter they get because every time cells divide, the telomeres get a tiny bit shorter. When they get too short, cells no longer reproduce. But in some reproductive cells and cancer cells, telomerase gene activity resets telomeres to the same length when DNA was originally copied, creating a kind of immortality for those cells.

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As 2021 draws to a close, COVID-19 remains a pervasive influence over life at Yale and across the world. And yet, even as a new surge in cases portends a winter of uncertainty, a look back at the past year offers many reminders of just how much more we now know about this global threat, the remarkable importance of vaccines and other public health measures, and the resiliency of humankind.

After reviewing the many hundreds of stories published on Yale News this year, we identified several about Yales response to the pandemic that especially resonated with readers and that best capture how the university and our experts have helped make sense of and respond to this disruptive disease. Youll find a list below.

In a second list below, we highlight several non-COVID stories about the people and projects that inspired us and gave us hope for a healthier and more equitable 2022 and the new initiatives that will position the Yale community to be a leader in tackling the challenges of the future.

As the new year began, Yale News reviewed how the campus community pulled together to do the work of the university in the face of unprecedented challenges, and looked ahead to the spring semester.

As chair of the White Houses COVID-19 Equity Task Force, Dr. Marcella Nunez-Smith, the C.N.H Long Professor of Internal Medicine, Public Health, and Management at Yale, became a national voice on racial inequities in COVID-19 treatment and outcomes. Meanwhile, Abbe Gluck, the Alfred M. Rankin Professor of Law and professor of internal medicine at Yale School of Medicine, was named special counsel to the Biden administrations COVID-19 Response team. She also worked in the Office of White House Counsel on other health care issues, including the Affordable Care Act..

In early January, Yale launched its COVID-19 vaccination program in the Lanman Center at Payne Whitney Gymnasium, as vaccines from Moderna and Pfizer gained final approvals for use in the United States.

After spending nearly a year cataloguing and exploring the SARS-CoV-2 genomes intricate makeup, a team of Yale scientists revealed a map of it with an unprecedented level of detail, including more than 100 identifiable structures.

In February, Yale scientists developed a new class of antiviral agents that showed promise for creating COVID-19 therapeutics exhibiting particular effectiveness when used in tandem with the drug remdesivir, another antiviral medication approved for use against the virus.

For most children, COVID-19 infection results in a relatively mild illness. In a few cases, however, a severe immune reaction occurs. During the spring, Yale research found that such rare, life-threatening reactions may be triggered by high levels of alarmins, molecules that make up part of the innate immune system.

The Lanman Center, which early in the pandemic was converted into a field hospital, and later into Yales primary vaccination center, returned to being simply a gym during the summer, as the vaccination operations were shifted to the Rose Center on Ashmun Street.

In July, a Yale-led study found that the COVID-19 vaccination campaign launched in the United States in late 2020 had, at that point, saved some 279,000 lives and prevented 1.25 million hospitalizations. Researchers warned, however, that these gains could be reversed by the highly transmissible Delta variant.

In September, Yale researchers provided important insights into what were then becoming known as breakthrough COVID-19 cases in which fully vaccinated individuals are infected by SARS-CoV-2 and which populations are particularly vulnerable to serious breakthrough illness.

Since the start of the COVID-19 pandemic, scientists had been unclear about how long immunity lasts after an unvaccinated person is infected. In October, a Yale-led team of researchers found an answer: Strong protection following natural infection is short-lived, lasting as little as three months or less.

In October, a Yale-led study found that two of the commonly used COVID-19 vaccines provide protection against multiple variants of the virus that causes the disease, including the highly infectious Delta variant. Their findings also showed that those infected with the virus prior to vaccination exhibit a more robust immune response to all variants than those who were uninfected and fully vaccinated.

In November, a study by Yale political scientists and public health experts found that, when it comes to persuading people to get vaccinated against COVID-19, its more effective to appeal to community spirit than to self-interest.

Breakthrough SARS-CoV-2 infections tend to be mild, but Yale research published in December showed that more older adults have developed severe breakthrough cases during the Delta variant phase of the pandemic, particularly after a longer period of time had elapsed since their last vaccination. The findings, researchers say, reveal the importance of booster vaccinations.

White evangelical Christians have resisted getting vaccinated against COVID-19 at higher rates than other religious groups in the U.S. In November, a Yale study found that persuading these vaccine holdouts had only become more difficult.

In December, as a new COVID-19 variant, Omicron, began to spread throughout the world, public health leaders scrambled to better understand how contagious the new variant is and whether existing vaccines are effective against it. Yale doctors offered insights into the emerging threat.

In February, Yale announced the establishment of the Wu Tsai Institute, an ambitious new research enterprise that will supercharge Yales neuroscience initiative and position the university to reveal the brain in its full, dynamic complexity, thanks to a historic gift from Joseph C. Tsai 86, 90 J.D. and his wife, Clara Wu Tsai.

As a historic renovation of the Peabody Museum proceeds, conservator Mariana Di Giacomo is charged with keeping a close eye on the iconic mural The Age of Reptiles, by celebrated artist Rudolph Zallinger. The experience has allowed her to appreciate layers of detail. In February, Yale News caught up with her and shared a dazzling gallery of images.

In a promising early trial, researchers from Yale reported in February that patients with spinal cord injuries experienced substantial improvements in motor function such as the ability to walk or to use their hands after an intravenous injection of bone marrow-derived stem cells.

After 30 months of renovations, the redesigned Humanities Quadrangle formerly the Hall of Graduate Studies put a vibrant new face on Yales longstanding excellence in the humanities. The refurbished building includes dynamic spaces that promote connections among departments and programs and the cultivation of new ideas.

The late Jeremy Ayers once known as the gender-bending performance artist Silva Thin may seem like an unlikely namesake for an ant. But thanks to Yale ecologist Douglas B. Booher and rock star Michael Stipe, who shared a decades-long friendship with Ayers, a new species from the forests of Ecuador will honor his legacy and his reverence for the diversity of life.

During the summer, the university announced that present and future students at Yale Universitys drama school will no longer pay tuition, thanks to a landmark $150 million gift from entertainment executive and philanthropist David Geffen.

Psilocybin, a psychedelic drug found in some mushrooms, has long been studied as a potential treatment for depression. Yale research published in July detailed exactly what happens in the brain after a dose of psilocybin, and what makes its medicinal properties so promising.

In August, Yale scientists published a study of atmospheric patterns on Mars and Saturns moon Titan that will help lay the foundation for more accurate forecasts of weather on other worlds. Researchers say such forecasts will be vital to the safety and success of future research missions.

In 1965, Yale scholars created a sensation with the revelation of the Vinland Map, which was thought to be the earliest known European depiction of the New World. This summer, a team of Yale researchers said it proved the map to be an elaborate 20th-century forgery.

In October, a series of performances by the Yale Glee Club, Yale Bands, and the Yale Symphony Orchestra held in each of Yales residential college courtyards marked a return to live music on campus following a year of lockdowns and a response to the Black Lives Matter protests of 2020. (With video.)

In November, Yale and the City of New Haven reconfirmed their historic, three-century partnership for a new generation, announcing a six-year commitment that increases the universitys annual voluntary financial contribution to the city and creates bold opportunities for inclusive economic growth that benefit the entire community.

Tony Reno, now in his ninth season as head coach of the Yale football team, is more focused on creating a culture of responsibility, camaraderie, and integrity than on wins and losses but that hasnt kept the Bulldogs from finding success on the field.

On the long road to Yale College, Obed Gyedu-Larbi labored as a domestic aide and Greyhound baggage handler. He also founded a non-profit to feed and clothe homeless people in New York City. For me, he said, it was important to not only work hard for myself.

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2021: The year in review | YaleNews - Yale News

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Las Vegas, USAPolymyositis Pipeline to Progress with New and Emerging Drugs for Treatment, Analyzes , Dec. 08, 2021 (GLOBE NEWSWIRE) -- DelveInsights Polymyositis Pipeline Insight 2021 report offers exhaustive global coverage of available, marketed, and pipeline therapies in different phases of clinical development, major pharmaceutical companies working to advance the pipeline space, and future growth potential of the Polymyositis pipeline domain.

Some of the essential takeaways from thePolymyositis Pipelinereport:

Request a sample and discover more about the report offerings @ Polymyositis Emerging Therapies

The Polymyositis pipeline report lays down detailed profiles of the pipeline assets, comparative analysis of clinical and non-clinical stage Polymyositis products, inactive and dormant assets, comprehensive assessment of driving and restraining factors, as well as the opportunities and risks in the Polymyositis pipeline landscape.

Polymyositis Overview

Polymyositis is a type of inflammatory myopathy, which refers to a group of muscle diseases characterized by chronic muscle inflammation and weakness. Polymyositis (PM), an autoimmune disorder, develops due to abnormal activation of cytotoxic T lymphocytes (CD8 cells) and macrophages against muscular antigens as well as the strong extrafusal muscular expression of major histocompatibility complex 1 causing damage to the endomysium of skeletal muscles. Polymyositis develops gradually over time, and it rarely affects persons younger than age 18.

Find out more about the disease and recent developments @Polymyositis Pipeline Assessment

Polymyositis Pipeline Drugs

Learn more about the novel and emerging Polymyositis pipeline therapies @ Polymyositis Pipeline Analysis

Polymyositis Therapeutics Assessment

ThePolymyositis Pipelinereport proffers an integral view of the Polymyositis emerging novel therapies segmented by Stage, Product Type, Molecule Type, Mechanism of Action and Route of Administration.

By Product Type

By Stage

By Route of Administration

By Molecule Type

By Mechanism of Action

Scope of the Polymyositis Pipeline Report

Dive deep into rich insights for emerging therapies and assessment, visit @ Polymyositis Emerging Therapies

Table of Contents

For further information on the Polymyositis current pipeline therapeutics, reach out @ Polymyositis Ongoing Clinical Trials

Track and assess a candidates clinical development journey through Actionable Intelligence and Comparative Therapeutic Assessment

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Polymyositis Pipeline to Progress with New and Emerging Drugs for Treatment, Analyzes DelveInsight - GlobeNewswire

Spinal Cord Stem Cells Comments Off on Polymyositis Pipeline to Progress with New and Emerging Drugs for Treatment, Analyzes DelveInsight – GlobeNewswire | December 10th, 2021

SEATTLE, Nov. 18, 2021 /PRNewswire/ -- According to Latest Report, The global cell and gene therapy marketis estimated to account for 47,095.2 Mn in terms of value by the end of 2028.

Genetic mutations can lead to a wide range of serious malfunctions at the cellular level, including diseases such as cancer. These treatments use "living drugs" to repair damaged tissues and replace diseased organs, and they have the potential to cure a wide variety of ailments. In addition to regenerating damaged organs, cell and gene therapy can cure cancer, and the treatment process is fast-paced, with significant progress made in recent years. For the cell and gene therapy industry to reach its full potential, early interaction with payers and regulators is crucial. This will facilitate a fast-tracked clinical trial. While embracing new platform technologies is challenging, early collaboration with other industries will ensure a faster path to market for the new therapies. In addition to this, a play-to-win attitude is critical to success in this field. The success of gene and cell therapies will depend on achieving clinical and research goals.

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Market Drivers

1. Increasing incidence of cancer and other target diseases is expected to drive growth of the global cell and gene therapy market during the forecast period

With growing incidence of cancer and target diseases such as measles and tuberculosis, the adoption of gene and cell therapy has increased. According to the World Health Organization (WHO), in 2019, around 1.4 million people died from tuberculosis worldwide with around 10 million people being diagnosed with the same. According to the same source, in 2018, around 9.6 million died due to cancer with over 300,000 new cases of cancer being diagnosed each year among children aged 0-19 years across the globe. Gene therapy uses genes to treat or prevent disease, where it allows doctors to insert a gene into a patient's cells instead of using drugs or surgery. Therefore, it has the potential to completely treat genetic disorders.

2. Growing investments in pharmaceutical R&D activities are expected to propel the global cell andgene therapy market growth over the forecast period

Key pharmaceutical companies in the market are focused on research and development activities pertaining to gene therapy. Currently, gene therapy is being widely researched for various diseases including cancer, cystic fibrosis, hemophilia, AIDS, and diabetes. For instance, in November 2021, Sio Gene Therapies reported positive interim data for gene therapy trial of Phase I/II of AXO-AAV-GM1 for the treatment of GM1 gangliosidosis, a genetic disorder that progressively destroys nerve cells in the brain and spinal cord.

Market Opportunity

1. Increasing demand for cell and gene therapies can present lucrative growth opportunities

The demand for cell and gene therapies is increasing with growing cases of genetic disorders, chronic diseases, etc. According to the Cystic Fibrosis Foundation (CFF), in the U.S., over 1,000 new cases of cystic fibrosis are diagnosed each year. Moreover, According to the WHO, the number of people with diabetes has increased from 108 million in 1980 to 422 million in 2014. According to the same source, in 2016, around 1.6 million deaths were directly caused due to diabetes. Cell and gene therapies have the potential to treat the aforementioned diseases.

2. Growing regulatory approval can provide major business opportunities

Key companies are focused on research and development activities, in order to gain regulatory approval and enhance market presence. For instance, in March 2021, Celgene Corporation, a subsidiary of Bristol Myers Squibb, received the U.S. Food and Drug Administration (FDA) approval for the first cell-based gene therapy Abecma indicated for the treatment of multiple myeloma.

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Market Trends

1. Stem cell therapy

In the recent past, stem cell therapies have gained significant importance across the healthcare sector. Stem cell therapy has the potential to treat tissue damage and have low immunogenicity. Furthermore, it can enhance the growth of new healthy skin tissues, improve collagen production, stimulate hair development after loss, and can be used in the treatment of various diseases including Parkinson's disease, Alzheimer's disease, cancer, spinal cord injury, etc.

2. North America Trends

Among regions, North America is expected to witness significant growth in the global cell and gene therapy market during the forecast period. This is owing to ongoing clinical trials combined with key companies focusing on R&D activities pertaining to cell and gene therapy. Moreover, the presence of key market players such as Thermo Fisher Scientific, Takara Bio Inc., Catalent Inc., and more are expected to boost the regional market growth in the near future.

Competitive Section

Major companies operating in the global cell and gene therapy market are Thermo Fisher Scientific, Merck KGaA, Lonza, Takara Bio Inc., Catalent Inc., F. Hoffmann-La Roche Ltd, Samsung Biologics, Wuxi Advanced Therapies, Boehringer Ingelheim, Novartis AG, and Miltenyi Biotec.

For instance, in July 2021, Minova Therapeutics Inc. entered into a collaboration and license agreement with Astellas Pharma Inc. for the research, development, and commercialization of novel cell therapy programs for diseases caused by mitochondrial dysfunction.

Global cell and gene therapy Market, By Region:

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Cell and Gene Therapy Market to reach US$ 47,095.2 Mn by end of 2028, Says Coherent Market Insights - PRNewswire

Spinal Cord Stem Cells Comments Off on Cell and Gene Therapy Market to reach US$ 47,095.2 Mn by end of 2028, Says Coherent Market Insights – PRNewswire | November 22nd, 2021

WHEN it comes to health, the news in recent times has been sombre.

It has been another rollercoaster year battling Covid, with the UK emerging from a third lockdown in spring.

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Millions of people have since had their jabs, and boosters are being rolled out as winter looms large.

But Covid is not the only big health story to come out of the past two years.

Behind the scenes, scientists around the world have been working on medical trials in the hope of finding cures for major illnesses.

And there have been dozens of major breakthroughs that could save billions of lives and change the way diseases are treated forever.

Just this month it emerged the vaccine for the human papillomavirus virus (HPV) could eradicate cervical cancer within the next few years.

From asthma to Alzheimers and cancer to infertility, CLARE OREILLY looks at the new treatments, vaccines and medicines that could put an end to some of the most common and deadly conditions.

CERVICAL cancer kills more than two women every day in the UK, claiming around 850 lives every year.

Yet a new study has found the disease could soon be a thing of the past.

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Kings College London scientists found the human papillomavirus (HPV) vaccine cut cases by 90 per cent.

The jab, which was first rolled out to teenage girls in the UK in 2008 then to boys in 2019, prevents HPV, which is responsible for nearly all cases of cervical cancer.

The study, in the Lancet, tracked women who received some of the first doses and found it prevented an estimated 17,200 pre-cancers and 450 cases in women in their twenties.

Cancer Research UKs chief executive Michelle Mitchell said: Its a historic moment to see the first study showing that the HPV vaccine has and will continue to protect women from cervical cancer.

A NEW antibody-based treatment developed by scientists in the UK and Germany could soon yield a vaccine to prevent Alzheimers.

The degenerative condition is thought to be caused by a type of protein that sticks to brain cells.

13

The scientists were able to trigger the immune system to make antibodies, which targeted the protein before it was deposited.

Professor Mark Carr, who led a team at the University of Leicester, said: It has the real potential to provide an effective treatment for Alzheimers using a therapeutic antibody and highlights the potential of a simple vaccine.

Meanwhile, a year-long study has started in Norway where Alzheimers patients will receive a transfusion of blood taken from runners.

It is hoped the chemicals released in the blood after running have a rejuvenating effect to slow disease progression.

THREE new drugs are being put through trials in the hope they could end the misery of hot flushes for menopausal women.

Hot flushes are thought to be caused by changes in hormone levels affecting the bodys temperature control.

13

But the medicines fezolinetant, elinzanetant and pavinetant can block the receptors which are responsible for the common symptom.

London GP Dr Zoe Watson says it could be years before the treatment is available on the NHS, though.

She says: It looks interesting in theory, but there are question marks over its efficacy, its side-effect profile and its cost.

Certainly if it does this well then it could be extremely useful for women whose most troubling menopausal symptom is hot flushes.

However, menopause is much more than just hot flushes and halting periods."

A BRAND new injection could reverse spinal cord injuries and allow patients to walk again just four weeks after treatment.

Developed by a team at Northwestern University in the US, the jab encourages nerves to regrow.

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It gave paralysed mice the ability to walk and human trials are expected to begin next year.

For decades, this has remained a major challenge for scientists because our bodys central nervous system, which includes the brain and spinal cord, doesnt have any significant capacity to repair itself after injury.

Professor Samuel Stupp said: Our research aims to find a therapy that can prevent individuals from becoming paralysed after major trauma or disease.

We are going straight to the FDA [the US Food and Drug Administration] to get this approved for use in patients.

DEMENTIA affects around 850,000 people in the UK and costs 26.3billion a year, but scientists at Durham University have made a breakthrough.

They are working on a treatment that could boost memory and muscle control in patients with the killer disease.

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Using infrared light to zap the brain improved the memory and thought processing in trials of healthy people.

And the next step is to enlist dementia patients to test the therapy.

Its delivered by a specially equipped helmet, which beams invisible light waves into the brain and forces cells to boost levels, improving blood flow too.

Dr Paul Chazot, who led the study, said: While more research is needed, there are promising signs that therapy involving infrared light might also be beneficial for people living with dementia and this is worth exploring.

ANYONE with asthma knows how debilitating it can be to receive a diagnosis.

Yet more than five million people in the UK are asthmatic. But a brand new drug, already approved for use on the NHS, is set to transform the lives of many with the condition, making attacks less frequent and less severe.

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Dupilumab is prescribed to treat eczema and rhinosinusitis a type of sinusitis where the nasal cavity as well as sinuses become inflamed.

Its from a family of drugs used to treat Covid.

Currently only patients with very serious asthma who have had at least four severe asthma attacks in the last year and are ineligible for other biological treatments will be considered for a prescription.

But the drug is set to change the lives of many asthma sufferers across the country.

HALF of us will get cancer at some point in our lives. But new jab Survivin could change the landscape dramatically, scientists say.

The first clinical trials are already under way, and the injection works to boost the bodys immune system. It supercharges the immune cells, helping them seek out and destroy cancerous cells while leaving healthy cells alone.

13

Currently there are 36 terminally ill patients taking part in the trials, which are focused on ovarian, prostate and lung cancers.

Michelle Mitchell, chief executive of Cancer Research UK, said: Just this month we heard the HPV vaccine has likely prevented hundreds of women from developing cervical cancer.

This is a new and exciting frontier in cancer medicine and if this trial and others are successful, we could see thousands more lives saved.

AROUND seven per cent of all men are affected by infertility.

And while treatments currently focus on solutions rather than cures, scientists at the University of Georgia, in the US, are looking to reverse male infertility altogether.

13

The researchers have used primate embryonic stem cells the building blocks of all cells in the body to grow sperm cells in the earlier stages of development in a petri dish.

These spermatids, which lack a head and tail for swimming, were capable of fertilising a rhesus macaque egg in vitro.

Lead researcher and associate professor Dr Charles Easley says: This is a major breakthrough towards producing stem cell-based therapies to treat male infertility in cases where the men do not produce any viable sperm cells.

It is the first step that shows this technology is potentially translatable.

GETTING through the blood/brain barrier to target treatments for brain cancer is complex.

But now a team of scientists in Toronto, Canada, have found a way to use ultrasound beams.

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They help open the barrier and can help facilitate drug delivery, which could change the way the disease is treated.

A trial this year saw four women with breast cancer that had spread to their brains treated with magnetic resonance-guided focused ultrasound (MRgFUS).

It allowed the antibody therapy herceptin to pass into their brain tissue, and caused the tumours to shrink without damaging any healthy tissue.

Dr Nir Lipsman, who led the study, said: It has long been theorised that focused ultrasound can be used to enhance drug delivery, but this is the first time we have shown we can get drugs into the brain.

A DRUG taken in pill form is to be trialled to combat the deadliest form of cancer.Auceliciclib is already used to treat brain tumours.

But now scientists hope it can help fight pancreatic cancer, which is often first diagnosed when it is at a late stage.

13

Professor Shudong Wang and her team at the University of South Australia are also working on new ways to detect the disease.

She said: Pancreatic cancer is extremely difficult to diagnose at an early stage because there are very few symptoms.

If it is caught early the malignant tumour can be surgically removed, but once it spreads into other organs it is lethal.

Chemotherapy and radiotherapy only buy patients a little extra time.

The team hopes the drug will be more effective and with fewer side-effects than current treatment options.

THE heroic scientists who developed the Covid vaccine did not stop there.

The team at the University of Oxford has also developed a malaria jab that will save billions of lives.

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A trial showed 77 per cent of volunteers who were vaccinated stayed malaria-free over the following 12 months.

More than 100 malaria vaccines have been developed in recent decades, but the Oxford jab is the first to have such a high success rate.

Halidou Tinto, professor of parasitology and the principal investigator on the trial, said: These are very exciting results showing unprecedented efficacy levels from a vaccine that has been well-tolerated in our trial programme.

We look forward to the upcoming phase III trial to demonstrate large-scale safety and efficacy data for a vaccine that is greatly needed.

A DRUG that repairs cancerous cells could revolutionise the way breast cancer is treated.

Patients given olaparib as part of a two-and-a-half year trial were 42 per cent less likely to see their cancer return.

13

There was also a 43 per cent dEcrease in the risk of the disease spreading.

Until the breakthrough earlier this year, the drug was mainly used for late-stage cancers, but the new findings suggest it is effective as an early treatment.

Professor Andrew Tutt, professor of oncology at the Institute of Cancer Research who led the study, said: Women with early-stage breast cancer who have inherited BRCA1 or BRCA2 mutations are typically diagnosed at a younger age.

Up to now, there has been no treatment that specifically targets the unique biology of these cancers to reduce the rate of recurrence, beyond initial treatment such as surgery.

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From asthma to cancer to infertility, the new treatments, jabs and meds making us healthier... - The Sun

Spinal Cord Stem Cells Comments Off on From asthma to cancer to infertility, the new treatments, jabs and meds making us healthier… – The Sun | November 22nd, 2021